                         SEQUENCE LISTING

<110>  The Board of Regents of the University of Texas System
       Gray, Steven
       Sinnett, Sarah
 
<120>  TRANSGENE CASSETTES DESIGNED TO EXPRESS A HUMAN MECP2 GENE

<130>  TAYS-003

<150>  US 62/944,209
<151>  2019-12-05

<150>  US 62/946,696
<151>  2019-12-11

<150>  US 63/008,159
<151>  2019-04-10

<150>  US 63/047,596
<151>  2019-07-02

<160>  30    

<170>  PatentIn version 3.5

<210>  1
<211>  181
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  miniMeCP2

<400>  1

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>  2
<211>  180
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  miniMeCP2

<400>  2

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


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


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


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


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


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


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


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


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


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


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


Arg Glu Thr Val 
            180 


<210>  3
<211>  543
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miniMeCP2

<400>  3
atggccgccg ccgccgccgc cgcgccgagc ggaggaggag gaggaggcga ggaggagaga       60

ctggaagaaa agccggctgt gccggaagct tctgcctccc ccaaacagcg gcgctccatc      120

atccgtgacc ggggacccat gtatgatgac cccaccctgc ctgaaggctg gacacggaag      180

cttaagcaaa ggaaatctgg ccgctctgct gggaagtatg atgtgtattt gatcaatccc      240

cagggaaaag cctttcgctc taaagtggag ttgattgcgt acttcgaaaa ggtaggcgac      300

acatccctgg accctaatga ttttgacttc acggtaactg ggagagggag cccctcccgg      360

cgagagcaga aaccacctgg atccagtggc agctctgggc ccaagaaaaa gcggaaggtg      420

ccggggagtg tggtggcagc cgctgccgcc gaggccaaaa agaaagccgt gaaggagtct      480

tctatccgat ctgtgcagga gaccgtactc cccatcaaga agcgcaagac ccgggagacg      540

gtc                                                                    543


<210>  4
<211>  540
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miniMeCP2

<400>  4
gccgccgccg ccgccgccgc gccgagcgga ggaggaggag gaggcgagga ggagagactg       60

gaagaaaagc cggctgtgcc ggaagcttct gcctccccca aacagcggcg ctccatcatc      120

cgtgaccggg gacccatgta tgatgacccc accctgcctg aaggctggac acggaagctt      180

aagcaaagga aatctggccg ctctgctggg aagtatgatg tgtatttgat caatccccag      240

ggaaaagcct ttcgctctaa agtggagttg attgcgtact tcgaaaaggt aggcgacaca      300

tccctggacc ctaatgattt tgacttcacg gtaactggga gagggagccc ctcccggcga      360

gagcagaaac cacctggatc cagtggcagc tctgggccca agaaaaagcg gaaggtgccg      420

gggagtgtgg tggcagccgc tgccgccgag gccaaaaaga aagccgtgaa ggagtcttct      480

atccgatctg tgcaggagac cgtactcccc atcaagaagc gcaagacccg ggagacggtc      540


<210>  5
<211>  10
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Myc Tag

<400>  5

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


<210>  6
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Myc Tag

<400>  6
gaacaaaaac tcatctcaga agaggatctg                                        30


<210>  7
<211>  7
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA binding site

<400>  7
ccaaaga                                                                  7


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

<220>
<223>  miRNA binding site

<400>  8
acttgaa                                                                  7


<210>  9
<211>  7
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA binding site

<400>  9
atgtgaa                                                                  7


<210>  10
<211>  7
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA binding site

<400>  10
agcacaa                                                                  7


<210>  11
<211>  7
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA binding site

<400>  11
ctgtgaa                                                                  7


<210>  12
<211>  8
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA binding site

<400>  12
ctacctca                                                                 8


<210>  13
<211>  175
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Reg2 sequence

<400>  13
ctgttctagc ccccaaagag ttttctgtgc ttgcttttga aacttgaagt cttgaaaacc       60

aaagacatag atgtgaaaat tttaggcagt gtaagctgat agcacaagtt ctggcgactc      120

acaattatgc tgtgaatttt acaaaaagaa gcagtaatct acctcagccg ataac           175


<210>  14
<211>  221
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  RDH1pA

<400>  14
agctcgctga tcagcctcac aagaataaag gcagctgttg tctcttcaga agtagctttg       60

cacttttcta aactaggaat atcaccagga ctgttactca atgtgtgctg caggaaagct      120

gatatattta aaaacaaaag gtgtaaccta tttattatat aaagagtttg ccttataaat      180

ttacataaaa atgtccgttt gtgtcttttg ttgtaaaaat c                          221


<210>  15
<211>  89
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Other miRNA binding sites found in RDH1pA

<400>  15
acaagaataa aggcagctgt tgtctcttca gaagtagctt tgcacttttc taaactagga       60

atatcaccag gactgttact caatgtgtg                                         89


<210>  16
<211>  110
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  MeCP2 downstream pA

<400>  16
gaaagcactg atatatttaa aaacaaaagg tgtaacctat ttattatata aagagtttgc       60

cttataaatt tacataaaaa tgtccgtttg tgtcttttgt tgtaaaaatc                 110


<210>  17
<211>  398
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Regulatory Sequence

<400>  17
agctcgctga tcagcctcac aagaataaag gcagctgttg tctcttcaga agtagctttg       60

cacttttcta aactaggaat atcaccagga ctgttactca atgtgtggtc gacctgttct      120

agcccccaaa gagttttctg tgcttgcttt tgaaacttga agtcttgaaa accaaagaca      180

tagatgtgaa aattttaggc agtgtaagct gatagcacaa gttctggcga ctcacaatta      240

tgctgtgaat tttacaaaaa gaagcagtaa tctacctcag ccgataacga aagcactgat      300

atatttaaaa acaaaaggtg taacctattt attatataaa gagtttgcct tataaattta      360

cataaaaatg tccgtttgtg tcttttgttg taaaaatc                              398


<210>  18
<211>  104
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' AAV ITR

<400>  18
tgcgcgctcg ctcgctcact gaggccgccc gggcaaagcc cgggcgtcgg gcgacctttg       60

gtcgcccggc ctcagtgagc gagcgagcgc gcagagaggg agtg                       104


<210>  19
<211>  106
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' AAV ITR

<400>  19
ctgcgcgctc gctcgctcac tgaggccgcc ggcccaaagg gccggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtgg                     106


<210>  20
<211>  130
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  3' AAV ITR

<400>  20
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag                                                             130


<210>  21
<211>  145
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  3' AAV ITR

<400>  21
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgccggc cctttgggcc ggcggcctca gtgagcgagc      120

gagcgcgcag agagggagtg gccaa                                            145


<210>  22
<211>  426
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  meP426 Promoter

<400>  22
ataggcgcca agagcctaga cttccttaag cgccagagtc cacaagggcc cagttaatcc       60

tcaacattca aatgctgccc acaaaaccag cccctctgtg ccctagccgc ctcttttttc      120

caagtgacag tagaactcca ccaatccgca gctgaatggg gtccgcctct tttccctgcc      180

taaacagaca ggaactcctg ccaattgagg gcgtcaccgc taaggctccg ccccagcctg      240

ggctccacaa ccaatgaagg gtaatctcga caaagagcaa ggggtggggc gcgggcgcgc      300

aggtgcagca gcacacaggc tggtcgggag ggcggggcgc gacgtctgcc gtgcggggtc      360

ccggcatcgg ttgcgcgcgc gctccctcct ctcggagaga gggctgtggt aaaacccgtc      420

cggaaa                                                                 426


<210>  23
<211>  164
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  JeT promoter

<400>  23
gggcggagtt agggcggagc caatcagcgt gcgccgttcc gaaagttgcc ttttatggct       60

gggcggagaa tgggcggtga acgccgatga ttatataagg acgcgccggg tgtggcacag      120

ctagttccgt cgcagccggg atttgggtcg cggttcttgt ttgt                       164


<210>  24
<211>  225
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  meP229 Promoter

<400>  24
caattgaggg cgtcaccgct aaggctccgc cccagcctgg gctccacaac caatgaaggg       60

taatctcgac aaagagcaag gggtggggcg cgggcgcgca ggtgcagcag cacacaggct      120

ggtcgggagg gcggggcgcg acgtctgccg tgcggggtcc cggcatcggt tgcgcgcgcg      180

ctccctcctc tcggagagag ggctgtggta aaacccgtcc ggaaa                      225


<210>  25
<211>  796
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CBh promoter

<400>  25
tacataactt acggtaaatg gcccgcctgg ctgaccgccc aacgaccccc gcccattgac       60

gtcaatagta acgccaatag ggactttcca ttgacgtcaa tgggtggagt atttacggta      120

aactgcccac ttggcagtac atcaagtgta tcatatgcca agtacgcccc ctattgacgt      180

caatgacggt aaatggcccg cctggcattg tgcccagtac atgaccttat gggactttcc      240

tacttggcag tacatctacg tattagtcat cgctattacc atggtcgagg tgagccccac      300

gttctgcttc actctcccca tctccccccc ctccccaccc ccaattttgt atttatttat      360

tttttaatta ttttgtgcag cgatgggggc gggggggggg ggggggcgcg cgccaggcgg      420

ggcggggcgg ggcgaggggc ggggcggggc gaggcggaga ggtgcggcgg cagccaatca      480

gagcggcgcg ctccgaaagt ttccttttat ggcgaggcgg cggcggcggc ggccctataa      540

aaagcgaagc gcgcggcggg cgggagtcgc tgcgacgctg ccttcgcccc gtgccccgct      600

ccgccgccgc ctcgcgccgc ccgccccggc tctgactgac cgcgttactc ccacaggtga      660

gcgggcggga cggcccttct cctccgggct gtaattagct gagcaagagg taagggttta      720

agggatggtt ggttggtggg gtattaatgt ttaattacct ggagcacctg cctgaaatca      780

ctttttttca ggttgg                                                      796


<210>  26
<211>  123
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SV40 PolyA sequence

<400>  26
tgtttattgc agcttataat ggttacaaat aaagcaatag catcacaaat ttcacaaata       60

aagcattttt ttcactgcat tctagttgtg gtttgtccaa actcatcaat gtatcttatc      120

atg                                                                    123


<210>  27
<211>  1682
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  self-comp MeP426-miniMECP2-myc-reg2-RDH1PA

<400>  27
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 agaaaagccg gctgtgccgg aagcttctgc ctcccccaaa cagcggcgct      660

ccatcatccg tgaccgggga cccatgtatg atgaccccac cctgcctgaa ggctggacac      720

ggaagcttaa gcaaaggaaa tctggccgct ctgctgggaa gtatgatgtg tatttgatca      780

atccccaggg aaaagccttt cgctctaaag tggagttgat tgcgtacttc gaaaaggtag      840

gcgacacatc cctggaccct aatgattttg acttcacggt aactgggaga gggagcccct      900

cccggcgaga gcagaaacca cctggatcca gtggcagctc tgggcccaag aaaaagcgga      960

aggtgccggg gagtgtggtg gcagccgctg ccgccgaggc caaaaagaaa gccgtgaagg     1020

agtcttctat ccgatctgtg caggagaccg tactccccat caagaagcgc aagacccggg     1080

agacggtcgg gagctccggc agttctggag aacaaaaact catctcagaa gaggatctgg     1140

tcgactagag ctcgctgatc agcctcacaa gaataaaggc agctgttgtc tcttcagaag     1200

tagctttgca cttttctaaa ctaggaatat caccaggact gttactcaat gtgtggtcga     1260

cctgttctag cccccaaaga gttttctgtg cttgcttttg aaacttgaag tcttgaaaac     1320

caaagacata gatgtgaaaa ttttaggcag tgtaagctga tagcacaagt tctggcgact     1380

cacaattatg ctgtgaattt tacaaaaaga agcagtaatc tacctcagcc gataacgaaa     1440

gcactgatat atttaaaaac aaaaggtgta acctatttat tatataaaga gtttgcctta     1500

taaatttaca taaaaatgtc cgtttgtgtc ttttgttgta aaaatcacgc gtaggaaccc     1560

ctagtgatgg agttggccac tccctctctg cgcgctcgct cgctcactga ggccgggcga     1620

ccaaaggtcg cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc     1680

ag                                                                    1682


<210>  28
<211>  545
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miniMECP2

<400>  28
ccatggccgc cgccgccgcc gccgcgccga gcggaggagg aggaggaggc gaggaggaga       60

gactggaaga aaagccggct gtgccggaag cttctgcctc ccccaaacag cggcgctcca      120

tcatccgtga ccggggaccc atgtatgatg accccaccct gcctgaaggc tggacacgga      180

agcttaagca aaggaaatct ggccgctctg ctgggaagta tgatgtgtat ttgatcaatc      240

cccagggaaa agcctttcgc tctaaagtgg agttgattgc gtacttcgaa aaggtaggcg      300

acacatccct ggaccctaat gattttgact tcacggtaac tgggagaggg agcccctccc      360

ggcgagagca gaaaccacct ggatccagtg gcagctctgg gcccaagaaa aagcggaagg      420

tgccggggag tgtggtggca gccgctgccg ccgaggccaa aaagaaagcc gtgaaggagt      480

cttctatccg atctgtgcag gagaccgtac tccccatcaa gaagcgcaag acccgggaga      540

cggtc                                                                  545


<210>  29
<211>  1660
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  rAAV Sequence

<400>  29
ctgcgcgctc gctcgctcac tgaggccgcc ggcccaaagg gccggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggagat ctgaattcgg      120

tacccatagg cgccaagagc ctagacttcc ttaagcgcca gagtccacaa gggcccagtt      180

aatcctcaac attcaaatgc tgcccacaaa accagcccct ctgtgcccta gccgcctctt      240

ttttccaagt gacagtagaa ctccaccaat ccgcagctga atggggtccg cctcttttcc      300

ctgcctaaac agacaggaac tcctgccaat tgagggcgtc accgctaagg ctccgcccca      360

gcctgggctc cacaaccaat gaagggtaat ctcgacaaag agcaaggggt ggggcgcggg      420

cgcgcaggtg cagcagcaca caggctggtc gggagggcgg ggcgcgacgt ctgccgtgcg      480

gggtcccggc atcggttgcg cgcgcgctcc ctcctctcgg agagagggct gtggtaaaac      540

ccgtccggaa accatggccg ccgccgccgc cgccgcgccg agcggaggag gaggaggagg      600

cgaggaggag agactggaag aaaagccggc tgtgccggaa gcttctgcct cccccaaaca      660

gcggcgctcc atcatccgtg accggggacc catgtatgat gaccccaccc tgcctgaagg      720

ctggacacgg aagcttaagc aaaggaaatc tggccgctct gctgggaagt atgatgtgta      780

tttgatcaat ccccagggaa aagcctttcg ctctaaagtg gagttgattg cgtacttcga      840

aaaggtaggc gacacatccc tggaccctaa tgattttgac ttcacggtaa ctgggagagg      900

gagcccctcc cggcgagagc agaaaccacc tggatccagt ggcagctctg ggcccaagaa      960

aaagcggaag gtgccgggga gtgtggtggc agccgctgcc gccgaggcca aaaagaaagc     1020

cgtgaaggag tcttctatcc gatctgtgca ggagaccgta ctccccatca agaagcgcaa     1080

gacccgggag acggtctaga gctcgctgat cagcctcaca agaataaagg cagctgttgt     1140

ctcttcagaa gtagctttgc acttttctaa actaggaata tcaccaggac tgttactcaa     1200

tgtgtggtcg acctgttcta gcccccaaag agttttctgt gcttgctttt gaaacttgaa     1260

gtcttgaaaa ccaaagacat agatgtgaaa attttaggca gtgtaagctg atagcacaag     1320

ttctggcgac tcacaattat gctgtgaatt ttacaaaaag aagcagtaat ctacctcagc     1380

cgataacgaa agcactgata tatttaaaaa caaaaggtgt aacctattta ttatataaag     1440

agtttgcctt ataaatttac ataaaaatgt ccgtttgtgt cttttgttgt aaaaatcacg     1500

cgtgcatgca gatctaggaa cccctagtga tggagttggc cactccctct ctgcgcgctc     1560

gctcgctcac tgaggccggg cgaccaaagg tcgcccgacg ccggcccttt gggccggcgg     1620

cctcagtgag cgagcgagcg cgcagagagg gagtggccaa                           1660


<210>  30
<211>  145
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' ITR Sequence

<400>  30
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgccggccc aaagggccgg cgtcgggcga cctttggtcg cccggcctca gtgagcgagc      120

gagcgcgcag agagggagtg gccaa                                            145


