                         SEQUENCE LISTING

<110>  The University Court of the University of Glasgow
       The University Court of the University of Edinburgh
 
<120>  MeCP2 expression cassettes

<130>  P254402WO

<150>  GB 1704704.4
<151>  2017-03-24

<150>  GB 1704722.6
<151>  2017-03-24

<160>  32    

<170>  PatentIn version 3.5

<210>  1
<211>  104
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  scAAV vector containing AAV-2 ITR

<400>  1
gcgcgctcgc tcgctcactg aggccgcccg ggcaaagccc gggcgtcggg cgacctttgg       60

tcgcccggcc tcagtgagcg agcgagcgcg cagagaggga gtgg                       104


<210>  2
<211>  104
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  scAAV vector containing AVV-2 ITRs

<400>  2
ccactccctc tctgcgcgct cgctcgctca ctgaggccgg gcgaccaaag gtcgcccgac       60

gcccgggctt tgcccgggcg gcctcagtga gcgagcgagc gcgc                       104


<210>  3
<211>  499
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Human MeCP2 protein isoform 1

<400>  3

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Ser Glu Asp Gln Asp Leu Gln Gly 
            20                  25                  30          


Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys Lys Asp Lys Lys 
        35                  40                  45              


Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro Ser Ala His His 
    50                  55                  60                  


Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr Ser Glu Gly Ser 
65                  70                  75                  80  


Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg 
                85                  90                  95      


Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu 
            100                 105                 110         


Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser 
        115                 120                 125             


Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe 
    130                 135                 140                 


Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr 
145                 150                 155                 160 


Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser 
                165                 170                 175     


Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro Lys Ser Pro Lys 
            180                 185                 190         


Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys Gly Ser Gly Thr 
        195                 200                 205             


Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln Val Lys Arg Val 
    210                 215                 220                 


Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met Pro Phe Gln Thr 
225                 230                 235                 240 


Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr Thr Ser Thr Gln 
                245                 250                 255     


Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys Ala Glu Ala Asp 
            260                 265                 270         


Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro Gly Ser Val Val 
        275                 280                 285             


Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser Ser 
    290                 295                 300                 


Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys Thr 
305                 310                 315                 320 


Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val Lys Pro Leu Leu 
                325                 330                 335     


Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu Lys Thr Cys Lys 
            340                 345                 350         


Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys Gly Arg Ser Ser 
        355                 360                 365             


Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His His His His His 
    370                 375                 380                 


Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro Pro Leu Pro Pro 
385                 390                 395                 400 


Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr Ser Pro Pro Glu 
                405                 410                 415     


Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu Lys Met Pro Arg 
            420                 425                 430         


Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu Pro Ala Lys Thr 
        435                 440                 445             


Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu Lys Tyr Lys His 
    450                 455                 460                 


Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser Ser Met Pro Arg 
465                 470                 475                 480 


Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro Val Thr Glu Arg 
                485                 490                 495     


Val Ser Ser 
            


<210>  4
<211>  487
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Human MeCP2 protein isoform 2

<400>  4

Met Val Ala Gly Met Leu Gly Leu Arg Glu Glu Lys Ser Glu Asp Gln 
1               5                   10                  15      


Asp Leu Gln Gly Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys 
            20                  25                  30          


Lys Asp Lys Lys Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro 
        35                  40                  45              


Ser Ala His His Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr 
    50                  55                  60                  


Ser Glu Gly Ser Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser 
65                  70                  75                  80  


Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp 
                85                  90                  95      


Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys 
            100                 105                 110         


Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln 
        115                 120                 125             


Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys 
    130                 135                 140                 


Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr 
145                 150                 155                 160 


Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro 
                165                 170                 175     


Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys 
            180                 185                 190         


Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln 
        195                 200                 205             


Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met 
    210                 215                 220                 


Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr 
225                 230                 235                 240 


Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys 
                245                 250                 255     


Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro 
            260                 265                 270         


Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val 
        275                 280                 285             


Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys 
    290                 295                 300                 


Lys Arg Lys Thr Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val 
305                 310                 315                 320 


Lys Pro Leu Leu Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu 
                325                 330                 335     


Lys Thr Cys Lys Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys 
            340                 345                 350         


Gly Arg Ser Ser Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His 
        355                 360                 365             


His His His His Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro 
    370                 375                 380                 


Pro Leu Pro Pro Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr 
385                 390                 395                 400 


Ser Pro Pro Glu Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu 
                405                 410                 415     


Lys Met Pro Arg Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu 
            420                 425                 430         


Pro Ala Lys Thr Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu 
        435                 440                 445             


Lys Tyr Lys His Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser 
    450                 455                 460                 


Ser Met Pro Arg Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro 
465                 470                 475                 480 


Val Thr Glu Arg Val Ser Ser 
                485         


<210>  5
<211>  102
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  methyl-CpG binding domain (MBD) of human MeCP protein

<400>  5

Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile 
1               5                   10                  15      


Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly 
            20                  25                  30          


Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys 
        35                  40                  45              


Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys 
    50                  55                  60                  


Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp 
65                  70                  75                  80  


Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg 
                85                  90                  95      


Arg Glu Gln Lys Pro Pro 
            100         


<210>  6
<211>  40
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  NCoR/SMRT Interaction Domain (NID) of human MeCP2

<400>  6

Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Ala Val 
1               5                   10                  15      


Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys 
            20                  25                  30          


Lys Arg Lys Thr Arg Glu Thr Val 
        35                  40  


<210>  7
<211>  17
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  native MeCP2 NLS

<400>  7

Arg Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg 
1               5                   10                  15      


Lys 
    


<210>  8
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  SV40 Large T antigen NLS

<400>  8

Pro Lys Lys Lys Arg Lys Val 
1               5           


<210>  9
<211>  475
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2

<400>  9

Ser Glu Asp Gln Asp Leu Gln Gly Leu Lys Asp Lys Pro Leu Lys Phe 
1               5                   10                  15      


Lys Lys Val Lys Lys Asp Lys Lys Glu Glu Lys Glu Gly Lys His Glu 
            20                  25                  30          


Pro Val Gln Pro Ser Ala His His Ser Ala Glu Pro Ala Glu Ala Gly 
        35                  40                  45              


Lys Ala Glu Thr Ser Glu Gly Ser Gly Ser Ala Pro Ala Val Pro Glu 
    50                  55                  60                  


Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly 
65                  70                  75                  80  


Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu 
                85                  90                  95      


Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu 
            100                 105                 110         


Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala 
        115                 120                 125             


Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp 
    130                 135                 140                 


Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro 
145                 150                 155                 160 


Pro Lys Lys Pro Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg 
                165                 170                 175     


Gly Arg Pro Lys Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr Ser 
            180                 185                 190         


Glu Gly Val Gln Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu 
        195                 200                 205             


Leu Val Lys Met Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu Gly 
    210                 215                 220                 


Gly Gly Ala Thr Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro Gly 
225                 230                 235                 240 


Arg Lys Arg Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg 
                245                 250                 255     


Gly Arg Lys Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys 
            260                 265                 270         


Lys Lys Ala Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val 
        275                 280                 285             


Leu Pro Ile Lys Lys Arg Lys Thr Arg Glu Thr Val Ser Ile Glu Val 
    290                 295                 300                 


Lys Glu Val Val Lys Pro Leu Leu Val Ser Thr Leu Gly Glu Lys Ser 
305                 310                 315                 320 


Gly Lys Gly Leu Lys Thr Cys Lys Ser Pro Gly Arg Lys Ser Lys Glu 
                325                 330                 335     


Ser Ser Pro Lys Gly Arg Ser Ser Ser Ala Ser Ser Pro Pro Lys Lys 
            340                 345                 350         


Glu His His His His His His His Ser Glu Ser Pro Lys Ala Pro Val 
        355                 360                 365             


Pro Leu Leu Pro Pro Leu Pro Pro Pro Pro Pro Glu Pro Glu Ser Ser 
    370                 375                 380                 


Glu Asp Pro Thr Ser Pro Pro Glu Pro Gln Asp Leu Ser Ser Ser Val 
385                 390                 395                 400 


Cys Lys Glu Glu Lys Met Pro Arg Gly Gly Ser Leu Glu Ser Asp Gly 
                405                 410                 415     


Cys Pro Lys Glu Pro Ala Lys Thr Gln Pro Ala Val Ala Thr Ala Ala 
            420                 425                 430         


Thr Ala Ala Glu Lys Tyr Lys His Arg Gly Glu Gly Glu Arg Lys Asp 
        435                 440                 445             


Ile Val Ser Ser Ser Met Pro Arg Pro Asn Arg Glu Glu Pro Val Asp 
    450                 455                 460                 


Ser Arg Thr Pro Val Thr Glu Arg Val Ser Ser 
465                 470                 475 


<210>  10
<211>  241
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 fragment designated Delta NC

<400>  10

Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile 
1               5                   10                  15      


Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly 
            20                  25                  30          


Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys 
        35                  40                  45              


Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys 
    50                  55                  60                  


Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp 
65                  70                  75                  80  


Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg 
                85                  90                  95      


Arg Glu Gln Lys Pro Pro Lys Lys Pro Lys Ser Pro Lys Ala Pro Gly 
            100                 105                 110         


Thr Gly Arg Gly Arg Gly Arg Pro Lys Gly Ser Gly Thr Thr Arg Pro 
        115                 120                 125             


Lys Ala Ala Thr Ser Glu Gly Val Gln Val Lys Arg Val Leu Glu Lys 
    130                 135                 140                 


Ser Pro Gly Lys Leu Leu Val Lys Met Pro Phe Gln Thr Ser Pro Gly 
145                 150                 155                 160 


Gly Lys Ala Glu Gly Gly Gly Ala Thr Thr Ser Thr Gln Val Met Val 
                165                 170                 175     


Ile Lys Arg Pro Gly Arg Lys Arg Lys Ala Glu Ala Asp Pro Gln Ala 
            180                 185                 190         


Ile Pro Lys Lys Arg Gly Arg Lys Pro Gly Ser Val Val Ala Ala Ala 
        195                 200                 205             


Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser Ser Ile Arg Ser 
    210                 215                 220                 


Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys Thr Arg Glu Thr 
225                 230                 235                 240 


Val 
    


<210>  11
<211>  157
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Protein variant designated Delta NIC

<400>  11

Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile 
1               5                   10                  15      


Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly 
            20                  25                  30          


Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys 
        35                  40                  45              


Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys 
    50                  55                  60                  


Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp 
65                  70                  75                  80  


Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg 
                85                  90                  95      


Arg Glu Gln Lys Pro Pro Gly Ser Ser Gly Ser Ser Gly Pro Lys Lys 
            100                 105                 110         


Lys Arg Lys Val Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala 
        115                 120                 125             


Lys Lys Lys Ala Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr 
    130                 135                 140                 


Val Leu Pro Ile Lys Lys Arg Lys Thr Arg Glu Thr Val 
145                 150                 155         


<210>  12
<211>  24
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  N-terminal portion

<400>  12

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys 
            20                  


<210>  13
<211>  12
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  N-terminal portion

<400>  13

Met Val Ala Gly Met Leu Gly Leu Arg Glu Glu Lys 
1               5                   10          


<210>  14
<211>  499
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette, human isoform 1

<400>  14

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Ser Glu Asp Gln Asp Leu Gln Gly 
            20                  25                  30          


Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys Lys Asp Lys Lys 
        35                  40                  45              


Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro Ser Ala His His 
    50                  55                  60                  


Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr Ser Glu Gly Ser 
65                  70                  75                  80  


Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg 
                85                  90                  95      


Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu 
            100                 105                 110         


Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser 
        115                 120                 125             


Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe 
    130                 135                 140                 


Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr 
145                 150                 155                 160 


Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser 
                165                 170                 175     


Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro Lys Ser Pro Lys 
            180                 185                 190         


Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys Gly Ser Gly Thr 
        195                 200                 205             


Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln Val Lys Arg Val 
    210                 215                 220                 


Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met Pro Phe Gln Thr 
225                 230                 235                 240 


Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr Thr Ser Thr Gln 
                245                 250                 255     


Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys Ala Glu Ala Asp 
            260                 265                 270         


Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro Gly Ser Val Val 
        275                 280                 285             


Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser Ser 
    290                 295                 300                 


Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys Thr 
305                 310                 315                 320 


Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val Lys Pro Leu Leu 
                325                 330                 335     


Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu Lys Thr Cys Lys 
            340                 345                 350         


Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys Gly Arg Ser Ser 
        355                 360                 365             


Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His His His His His 
    370                 375                 380                 


Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro Pro Leu Pro Pro 
385                 390                 395                 400 


Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr Ser Pro Pro Glu 
                405                 410                 415     


Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu Lys Met Pro Arg 
            420                 425                 430         


Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu Pro Ala Lys Thr 
        435                 440                 445             


Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu Lys Tyr Lys His 
    450                 455                 460                 


Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser Ser Met Pro Arg 
465                 470                 475                 480 


Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro Val Thr Glu Arg 
                485                 490                 495     


Val Ser Ser 
            


<210>  15
<211>  487
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette, human isoform 2

<400>  15

Met Val Ala Gly Met Leu Gly Leu Arg Glu Glu Lys Ser Glu Asp Gln 
1               5                   10                  15      


Asp Leu Gln Gly Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys 
            20                  25                  30          


Lys Asp Lys Lys Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro 
        35                  40                  45              


Ser Ala His His Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr 
    50                  55                  60                  


Ser Glu Gly Ser Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser 
65                  70                  75                  80  


Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp 
                85                  90                  95      


Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys 
            100                 105                 110         


Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln 
        115                 120                 125             


Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys 
    130                 135                 140                 


Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr 
145                 150                 155                 160 


Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro 
                165                 170                 175     


Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys 
            180                 185                 190         


Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln 
        195                 200                 205             


Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met 
    210                 215                 220                 


Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr 
225                 230                 235                 240 


Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys 
                245                 250                 255     


Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro 
            260                 265                 270         


Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val 
        275                 280                 285             


Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys 
    290                 295                 300                 


Lys Arg Lys Thr Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val 
305                 310                 315                 320 


Lys Pro Leu Leu Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu 
                325                 330                 335     


Lys Thr Cys Lys Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys 
            340                 345                 350         


Gly Arg Ser Ser Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His 
        355                 360                 365             


His His His His Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro 
    370                 375                 380                 


Pro Leu Pro Pro Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr 
385                 390                 395                 400 


Ser Pro Pro Glu Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu 
                405                 410                 415     


Lys Met Pro Arg Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu 
            420                 425                 430         


Pro Ala Lys Thr Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu 
        435                 440                 445             


Lys Tyr Lys His Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser 
    450                 455                 460                 


Ser Met Pro Arg Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro 
465                 470                 475                 480 


Val Thr Glu Arg Val Ser Ser 
                485         


<210>  16
<211>  265
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette Delta NC isoform
       1

<400>  16

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Pro Ala Val Pro Glu Ala Ser Ala 
            20                  25                  30          


Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr 
        35                  40                  45              


Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg 
    50                  55                  60                  


Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro 
65                  70                  75                  80  


Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu 
                85                  90                  95      


Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val 
            100                 105                 110         


Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys 
        115                 120                 125             


Pro Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro 
    130                 135                 140                 


Lys Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val 
145                 150                 155                 160 


Gln Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys 
                165                 170                 175     


Met Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala 
            180                 185                 190         


Thr Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg 
        195                 200                 205             


Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys 
    210                 215                 220                 


Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala 
225                 230                 235                 240 


Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile 
                245                 250                 255     


Lys Lys Arg Lys Thr Arg Glu Thr Val 
            260                 265 


<210>  17
<211>  253
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette  Delta NC 
       isoform 2

<400>  17

Met Val Ala Gly Met Leu Gly Leu Arg Glu Glu Lys Pro Ala Val Pro 
1               5                   10                  15      


Glu Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg 
            20                  25                  30          


Gly Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys 
        35                  40                  45              


Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr 
    50                  55                  60                  


Leu Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile 
65                  70                  75                  80  


Ala Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe 
                85                  90                  95      


Asp Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys 
            100                 105                 110         


Pro Pro Lys Lys Pro Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly 
        115                 120                 125             


Arg Gly Arg Pro Lys Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr 
    130                 135                 140                 


Ser Glu Gly Val Gln Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys 
145                 150                 155                 160 


Leu Leu Val Lys Met Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu 
                165                 170                 175     


Gly Gly Gly Ala Thr Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro 
            180                 185                 190         


Gly Arg Lys Arg Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys 
        195                 200                 205             


Arg Gly Arg Lys Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala 
    210                 215                 220                 


Lys Lys Lys Ala Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr 
225                 230                 235                 240 


Val Leu Pro Ile Lys Lys Arg Lys Thr Arg Glu Thr Val 
                245                 250             


<210>  18
<211>  181
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette, Delta NIC 
       isoform 1

<400>  18

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Pro Ala Val Pro Glu Ala Ser Ala 
            20                  25                  30          


Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr 
        35                  40                  45              


Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg 
    50                  55                  60                  


Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro 
65                  70                  75                  80  


Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu 
                85                  90                  95      


Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val 
            100                 105                 110         


Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Gly Ser 
        115                 120                 125             


Ser Gly Ser Ser Gly Pro Lys Lys Lys Arg Lys Val Pro Gly Ser Val 
    130                 135                 140                 


Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser 
145                 150                 155                 160 


Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys 
                165                 170                 175     


Thr Arg Glu Thr Val 
            180     


<210>  19
<211>  169
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein encoded by the expression cassette, Delta NIC 
       isoform 2

<400>  19

Met Val Ala Gly Met Leu Gly Leu Arg Glu Glu Lys Pro Ala Val Pro 
1               5                   10                  15      


Glu Ala Ser Ala Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg 
            20                  25                  30          


Gly Pro Met Tyr Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys 
        35                  40                  45              


Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr 
    50                  55                  60                  


Leu Ile Asn Pro Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile 
65                  70                  75                  80  


Ala Tyr Phe Glu Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe 
                85                  90                  95      


Asp Phe Thr Val Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys 
            100                 105                 110         


Pro Pro Gly Ser Ser Gly Ser Ser Gly Pro Lys Lys Lys Arg Lys Val 
        115                 120                 125             


Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala 
    130                 135                 140                 


Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile 
145                 150                 155                 160 


Lys Lys Arg Lys Thr Arg Glu Thr Val 
                165                 


<210>  20
<211>  10
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  C-terminal c-Myc epitope tag

<400>  20

Glu Gln Lys Leu Ile Ser Glu Glu Asp Leu 
1               5                   10  


<210>  21
<211>  515
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MeCP2 protein comprising a heterologous sequence

<400>  21

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Ser Glu Asp Gln Asp Leu Gln Gly 
            20                  25                  30          


Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys Lys Asp Lys Lys 
        35                  40                  45              


Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro Ser Ala His His 
    50                  55                  60                  


Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr Ser Glu Gly Ser 
65                  70                  75                  80  


Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg 
                85                  90                  95      


Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu 
            100                 105                 110         


Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser 
        115                 120                 125             


Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe 
    130                 135                 140                 


Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr 
145                 150                 155                 160 


Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser 
                165                 170                 175     


Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro Lys Ser Pro Lys 
            180                 185                 190         


Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys Gly Ser Gly Thr 
        195                 200                 205             


Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln Val Lys Arg Val 
    210                 215                 220                 


Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met Pro Phe Gln Thr 
225                 230                 235                 240 


Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr Thr Ser Thr Gln 
                245                 250                 255     


Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys Ala Glu Ala Asp 
            260                 265                 270         


Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro Gly Ser Val Val 
        275                 280                 285             


Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser Ser 
    290                 295                 300                 


Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys Thr 
305                 310                 315                 320 


Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val Lys Pro Leu Leu 
                325                 330                 335     


Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu Lys Thr Cys Lys 
            340                 345                 350         


Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys Gly Arg Ser Ser 
        355                 360                 365             


Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His His His His His 
    370                 375                 380                 


Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro Pro Leu Pro Pro 
385                 390                 395                 400 


Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr Ser Pro Pro Glu 
                405                 410                 415     


Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu Lys Met Pro Arg 
            420                 425                 430         


Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu Pro Ala Lys Thr 
        435                 440                 445             


Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu Lys Tyr Lys His 
    450                 455                 460                 


Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser Ser Met Pro Arg 
465                 470                 475                 480 


Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro Val Thr Glu Arg 
                485                 490                 495     


Val Ser Ser Arg Gly Pro Phe Glu Gln Lys Leu Ile Ser Glu Glu Asp 
            500                 505                 510         


Leu Val Asp 
        515 


<210>  22
<211>  284
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  A c-Myc-tagged version of the Delta NC protein

<400>  22

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Pro Ala Val Pro Glu Ala Ser Ala 
            20                  25                  30          


Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr 
        35                  40                  45              


Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg 
    50                  55                  60                  


Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro 
65                  70                  75                  80  


Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu 
                85                  90                  95      


Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val 
            100                 105                 110         


Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys 
        115                 120                 125             


Pro Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro 
    130                 135                 140                 


Lys Gly Ser Gly Thr Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val 
145                 150                 155                 160 


Gln Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys 
                165                 170                 175     


Met Pro Phe Gln Thr Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala 
            180                 185                 190         


Thr Thr Ser Thr Gln Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg 
        195                 200                 205             


Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys 
    210                 215                 220                 


Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala 
225                 230                 235                 240 


Val Lys Glu Ser Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile 
                245                 250                 255     


Lys Lys Arg Lys Thr Arg Glu Thr Val Gly Ser Ser Gly Ser Ser Gly 
            260                 265                 270         


Glu Gln Lys Leu Ile Ser Glu Glu Asp Leu Val Asp 
        275                 280                 


<210>  23
<211>  200
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  c-Myc-tagged version of the Delta NIC protein

<400>  23

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Pro Ala Val Pro Glu Ala Ser Ala 
            20                  25                  30          


Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr 
        35                  40                  45              


Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg 
    50                  55                  60                  


Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro 
65                  70                  75                  80  


Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu 
                85                  90                  95      


Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val 
            100                 105                 110         


Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Gly Ser 
        115                 120                 125             


Ser Gly Ser Ser Gly Pro Lys Lys Lys Arg Lys Val Pro Gly Ser Val 
    130                 135                 140                 


Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser 
145                 150                 155                 160 


Ser Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys 
                165                 170                 175     


Thr Arg Glu Thr Val Gly Ser Ser Gly Ser Ser Gly Glu Gln Lys Leu 
            180                 185                 190         


Ile Ser Glu Glu Asp Leu Val Asp 
        195                 200 


<210>  24
<211>  130
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  5' transcriptional control comprising a core promoter element

<400>  24

Ala Ala Ala Cys Cys Ala Gly Cys Cys Cys Cys Thr Cys Thr Gly Thr 
1               5                   10                  15      


Gly Cys Cys Cys Thr Ala Gly Cys Cys Gly Cys Cys Thr Cys Thr Thr 
            20                  25                  30          


Thr Thr Thr Thr Cys Cys Ala Ala Gly Thr Gly Ala Cys Ala Gly Thr 
        35                  40                  45              


Ala Gly Ala Ala Cys Thr Cys Cys Ala Cys Cys Ala Ala Thr Cys Cys 
    50                  55                  60                  


Gly Cys Ala Gly Cys Thr Gly Ala Ala Thr Gly Gly Gly Gly Thr Cys 
65                  70                  75                  80  


Cys Gly Cys Cys Thr Cys Thr Thr Thr Thr Cys Cys Cys Thr Gly Cys 
                85                  90                  95      


Cys Thr Ala Ala Ala Cys Ala Gly Ala Cys Ala Gly Gly Ala Ala Cys 
            100                 105                 110         


Thr Cys Cys Thr Gly Cys Cys Ala Ala Thr Thr Gly Ala Gly Gly Gly 
        115                 120                 125             


Cys Gly 
    130 


<210>  25
<211>  62
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' transcriptional control region comprising a silencer element

<400>  25
ttaagcgcca gagtccacaa gggcccagtt aatcctcaac attcaaatgc tgcccacaaa       60

ac                                                                      62


<210>  26
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' transcriptional control region comprising a CNS regulatory 
       element

<400>  26
cagcacacag gctggtcgg                                                    19


<210>  27
<211>  29
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  a miR-22 binding site

<400>  27
acaagaataa aggcagctgt tgtctcttc                                         29


<210>  28
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  a miR-19 binding site

<400>  28
agaagtagct ttgcactttt ctaaactagg                                        30


<210>  29
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  a miR-132 binding site

<400>  29
aatatcacca ggactgttac tcaatgtgtg                                        30


<210>  30
<211>  13
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  3'UTR of the MeCP2 gene,  AU-rich element

<400>  30
auauauuuaa aaa                                                          13


<210>  31
<211>  23
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  MeCP2 gene, GU-rich region

<400>  31
uguccguuug ugucuuuugu ugu                                               23


<210>  32
<211>  6043
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  2nd generation expression cassette

<400>  32
tgcgcgctcg ctcgctcact gaggccgccc gggcaaagcc cgggcgtcgg gcgacctttg       60

gtcgcccggc ctcagtgagc gagcgagcgc gcagagaggg agtggggttc ggtacccata      120

ggcgccaaga gcctagactt ccttaagcgc cagagtccac aagggcccag ttaatcctca      180

acattcaaat gctgcccaca aaaccagccc ctctgtgccc tagccgcctc ttttttccaa      240

gtgacagtag aactccacca atccgcagct gaatggggtc cgcctctttt ccctgcctaa      300

acagacagga actcctgcca attgagggcg tcaccgctaa ggctccgccc cagcctgggc      360

tccacaacca atgaagggta atctcgacaa agagcaaggg gtggggcgcg ggcgcgcagg      420

tgcagcagca cacaggctgg tcgggagggc ggggcgcgac gtctgccgtg cggggtcccg      480

gcatcggttg cgcgcgcgct ccctcctctc ggagagaggg ctgtggtaaa acccgtccgg      540

aaaccatggc cgccgccgcc gccgccgcgc cgagcggagg aggaggagga ggcgaggagg      600

agagactgga agaaaagtca gaagaccagg acctccaggg cctcaaggac aaacccctca      660

agtttaaaaa ggtgaagaaa gataagaaag aagagaaaga gggcaagcat gagcccgtgc      720

agccatcagc ccaccactct gctgagcccg cagaggcagg caaagcagag acatcagaag      780

ggtcaggctc cgccccggct gtgccggaag cttctgcctc ccccaaacag cggcgctcca      840

tcatccgtga ccggggaccc atgtatgatg accccaccct gcctgaaggc tggacacgga      900

agcttaagca aaggaaatct ggccgctctg ctgggaagta tgatgtgtat ttgatcaatc      960

cccagggaaa agcctttcgc tctaaagtgg agttgattgc gtacttcgaa aaggtaggcg     1020

acacatccct ggaccctaat gattttgact tcacggtaac tgggagaggg agcccctccc     1080

ggcgagagca gaaaccacct aagaagccca aatctcccaa agctccagga actggcagag     1140

gccggggacg ccccaaaggg agcggcacca cgagacccaa ggcggccacg tcagagggtg     1200

tgcaggtgaa aagggtcctg gagaaaagtc ctgggaagct ccttgtcaag atgccttttc     1260

aaacttcgcc agggggcaag gctgaggggg gtggggccac cacatccacc caggtcatgg     1320

tgatcaaacg ccccggcagg aagcgaaaag ctgaggccga ccctcaggcc attcccaaga     1380

aacggggccg aaagccgggg agtgtggtgg cagccgctgc cgccgaggcc aaaaagaaag     1440

ccgtgaagga gtcttctatc cgatctgtgc aggagaccgt actccccatc aagaagcgca     1500

agacccggga gacggtcagc atcgaggtca aggaagtggt gaagcccctg ctggtgtcca     1560

ccctcggtga gaagagcggg aaaggactga agacctgtaa gagccctggg cggaaaagca     1620

aggagagcag ccccaagggg cgcagcagca gcgcctcctc accccccaag aaggagcacc     1680

accaccatca ccaccactca gagtccccaa aggcccccgt gccactgctc ccacccctgc     1740

ccccacctcc acctgagccc gagagctccg aggaccccac cagcccccct gagccccagg     1800

acttgagcag cagcgtctgc aaagaggaga agatgcccag aggaggctca ctggagagcg     1860

acggctgccc caaggagcca gctaagactc agcccgcggt tgccaccgcc gccacggccg     1920

cagaaaagta caaacaccga ggggagggag agcgcaaaga cattgtttca tcctccatgc     1980

caaggccaaa cagagaggag cctgtggaca gccggacgcc cgtgaccgag agagttagct     2040

ctagagggcc cttcgaacaa aaactcatct cagaagagga tctggtcgac tagagctcgc     2100

tgatcagcct cacaagaata aaggcagctg ttgtctcttc agaagtagct ttgcactttt     2160

ctaaactagg aatatcacca ggactgttac tcaatgtgtg ggtaccgaaa gcactgatat     2220

atttaaaaac aaaaggtgta acctatttat tatataaaga gtttgcctta taaatttaca     2280

taaaaatgtc cgtttgtgtc ttttgttgta aaaatcacgc gtaggaaccc ctagtgatgg     2340

agttggccac tccctctctg cgcgctcgct cgctcactga ggccgggcga ccaaaggtcg     2400

cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc agctggcgta     2460

atagcgaaga ggcccgcacc gatcgccctt cccaacagtt gcgcagcctg aatggcgaat     2520

ggcgattccg ttgcaatggc tggcggtaat attgttctgg atattaccag caaggccgat     2580

agtttgagtt cttctactca ggcaagtgat gttattacta atcaaagaag tattgcgaca     2640

acggttaatt tgcgtgatgg acagactctt ttactcggtg gcctcactga ttataaaaac     2700

acttctcagg attctggcgt accgttcctg tctaaaatcc ctttaatcgg cctcctgttt     2760

agctcccgct ctgattctaa cgaggaaagc acgttatacg tgctcgtcaa agcaaccata     2820

gtacgcgccc tgtagcggcg cattaagcgc ggcgggtgtg gtggttacgc gcagcgtgac     2880

cgctacactt gccagcgccc tagcgcccgc tcctttcgct ttcttccctt cctttctcgc     2940

cacgttcgcc ggctttcccc gtcaagctct aaatcggggg ctccctttag ggttccgatt     3000

tagtgcttta cggcacctcg accccaaaaa acttgattag ggtgatggtt cacgtagtgg     3060

gccatcgccc tgatagacgg tttttcgccc tttgacgttg gagtccacgt tctttaatag     3120

tggactcttg ttccaaactg gaacaacact caaccctatc tcggtctatt cttttgattt     3180

ataagggatt ttgccgattt cggcctattg gttaaaaaat gagctgattt aacaaaaatt     3240

taacgcgaat tttaacaaaa tattaacgct tacaatttaa atatttgctt atacaatctt     3300

cctgtttttg gggcttttct gattatcaac cggggtacat atgattgaca tgctagtttt     3360

acgattaccg ttcatcgatt ctcttgtttg ctccagactc tcaggcaatg acctgatagc     3420

ctttgtagag acctctcaaa aatagctacc ctctccggca tgaatttatc agctagaacg     3480

gttgaatatc atattgatgg tgatttgact gtctccggcc tttctcaccc gtttgaatct     3540

ttacctacac attactcagg cattgcattt aaaatatatg agggttctaa aaatttttat     3600

ccttgcgttg aaataaaggc ttctcccgca aaagtattac agggtcataa tgtttttggt     3660

acaaccgatt tagctttatg ctctgaggct ttattgctta attttgctaa ttctttgcct     3720

tgcctgtatg atttattgga tgttggaatt cctgatgcgg tattttctcc ttacgcatct     3780

gtgcggtatt tcacaccgca tatggtgcac tctcagtaca atctgctctg atgccgcata     3840

gttaagccag ccccgacacc cgccaacacc cgctgacgcg ccctgacggg cttgtctgct     3900

cccggcatcc gcttacagac aagctgtgac cgtctccggg agctgcatgt gtcagaggtt     3960

ttcaccgtca tcaccgaaac gcgcgagacg aaagggcctc gtgatacgcc tatttttata     4020

ggttaatgtc atgataataa tggtttctta gacgtcaggt ggcacttttc ggggaaatgt     4080

gcgcggaacc cctatttgtt tatttttcta aatacattca aatatgtatc cgctcatgag     4140

acaataaccc tgataaatgc ttcaataata ttgaaaaagg aagagtatga gtattcaaca     4200

tttccgtgtc gcccttattc ccttttttgc ggcattttgc cttcctgttt ttgctcaccc     4260

agaaacgctg gtgaaagtaa aagatgctga agatcagttg ggtgcacgag tgggttacat     4320

cgaactggat ctcaacagcg gtaagatcct tgagagtttt cgccccgaag aacgttttcc     4380

aatgatgagc acttttaaag ttctgctatg tggcgcggta ttatcccgta ttgacgccgg     4440

gcaagagcaa ctcggtcgcc gcatacacta ttctcagaat gacttggttg agtactcacc     4500

agtcacagaa aagcatctta cggatggcat gacagtaaga gaattatgca gtgctgccat     4560

aaccatgagt gataacactg cggccaactt acttctgaca acgatcggag gaccgaagga     4620

gctaaccgct tttttgcaca acatggggga tcatgtaact cgccttgatc gttgggaacc     4680

ggagctgaat gaagccatac caaacgacga gcgtgacacc acgatgcctg tagcaatggc     4740

aacaacgttg cgcaaactat taactggcga actacttact ctagcttccc ggcaacaatt     4800

aatagactgg atggaggcgg ataaagttgc aggaccactt ctgcgctcgg cccttccggc     4860

tggctggttt attgctgata aatctggagc cggtgagcgt gggtctcgcg gtatcattgc     4920

agcactgggg ccagatggta agccctcccg tatcgtagtt atctacacga cggggagtca     4980

ggcaactatg gatgaacgaa atagacagat cgctgagata ggtgcctcac tgattaagca     5040

ttggtaactg tcagaccaag tttactcata tatactttag attgatttaa aacttcattt     5100

ttaatttaaa aggatctagg tgaagatcct ttttgataat ctcatgacca aaatccctta     5160

acgtgagttt tcgttccact gagcgtcaga ccccgtagaa aagatcaaag gatcttcttg     5220

agatcctttt tttctgcgcg taatctgctg cttgcaaaca aaaaaaccac cgctaccagc     5280

ggtggtttgt ttgccggatc aagagctacc aactcttttt ccgaaggtaa ctggcttcag     5340

cagagcgcag ataccaaata ctgtccttct agtgtagccg tagttaggcc accacttcaa     5400

gaactctgta gcaccgccta catacctcgc tctgctaatc ctgttaccag tggctgctgc     5460

cagtggcgat aagtcgtgtc ttaccgggtt ggactcaaga cgatagttac cggataaggc     5520

gcagcggtcg ggctgaacgg ggggttcgtg cacacagccc agcttggagc gaacgaccta     5580

caccgaactg agatacctac agcgtgagct atgagaaagc gccacgcttc ccgaagggag     5640

aaaggcggac aggtatccgg taagcggcag ggtcggaaca ggagagcgca cgagggagct     5700

tccaggggga aacgcctggt atctttatag tcctgtcggg tttcgccacc tctgacttga     5760

gcgtcgattt ttgtgatgct cgtcaggggg gcggagccta tggaaaaacg ccagcaacgc     5820

ggccttttta cggttcctgg ccttttgctg gccttttgct cacatgttct ttcctgcgtt     5880

atcccctgat tctgtggata accgtattac cgcctttgag tgagctgata ccgctcgccg     5940

cagccgaacg accgagcgca gcgagtcagt gagcgaggaa gcggaagagc gcccaatacg     6000

caaaccgcct ctccccgcgc gttggccgat tcattaatgc agc                       6043


