                         SEQUENCE LISTING

<110>  Royal Holloway and Bedford New College
 
<120>  Neurodegenerative Disorders

<130>  88677PCT1

<150>  1803010.6
<151>  2018-02-26

<160>  28    

<170>  PatentIn version 3.5

<210>  1
<211>  168
<212>  DNA
<213>  Homo sapiens

<400>  1
aggtgtgctc tgaacaagcc gagacggggc cgtgccgagc aatgatctcc cgctggtact       60

ttgatgtgac tgaagggaag tgtgccccat tcttttacgg cggatgtggc ggcaaccgga      120

acaactttga cacagaagag tactgcatgg ccgtgtgtgg cagcgcca                   168


<210>  2
<211>  57
<212>  PRT
<213>  Homo sapiens

<400>  2

Glu Val Cys Ser Glu Gln Ala Glu Thr Gly Pro Cys Arg Ala Met Ile 
1               5                   10                  15      


Ser Arg Trp Tyr Phe Asp Val Thr Glu Gly Lys Cys Ala Pro Phe Phe 
            20                  25                  30          


Tyr Gly Gly Cys Gly Gly Asn Arg Asn Asn Phe Asp Thr Glu Glu Tyr 
        35                  40                  45              


Cys Met Ala Val Cys Gly Ser Ala Met 
    50                  55          


<210>  3
<211>  57
<212>  DNA
<213>  Homo sapiens

<400>  3
tgtcccaaag tttactcaag actacccagg aacctcttgc ccgagatcct gttaaac          57


<210>  4
<211>  20
<212>  PRT
<213>  Homo sapiens

<400>  4

Met Ser Gln Ser Leu Leu Lys Thr Thr Gln Glu Pro Leu Ala Arg Asp 
1               5                   10                  15      


Pro Val Lys Leu 
            20  


<210>  5
<211>  233
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Exon 7 target squence

<400>  5
atctcctctg attagaggtg tgctctgaac aagccgagac ggggccgtgc cgagcaatga       60

tctcccgctg gtactttgat gtgactgaag ggaagtgtgc cccattcttt tacggcggat      120

gtggcggcaa ccggaacaac tttgacacag aagagtactg catggccgtg tgtggcagcg      180

ccagtaagtg gacccttctt cgagcctggc cacctttcgt ctctctcgcc act             233


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

<220>
<223>  PMO 7.1

<400>  6
acggccccgt ctcggcttgt tcagagcaca                                        30


<210>  7
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 7.2

<400>  7
cacacttccc ttcagtcaca tcaaagtacc                                        30


<210>  8
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 7.4

<400>  8
ttgttccggt tgccgccaca tccgccgtaa                                        30


<210>  9
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 7.4

<400>  9
acggccatgc agtactcttc tgtgtcaaag                                        30


<210>  10
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 7.5

<400>  10
caggctcgaa gaagggtcca cttactggcg                                        30


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

<220>
<223>  AON target region of Exon 8 of App gene

<400>  11
cccatgtttc tctttttgtt tttagttatg ttctcttatt ttttccatag tgtcccaaag       60

tttactcaag actacccagg aacctcttgc ccgagatcct gttaaacgta cgttgtcatt      120

cacctgaggg aagggaagag gggaggagga tgctgct                               157


<210>  12
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 8.1

<400>  12
aaactttggg acactatgga aaaaa                                             25


<210>  13
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 8.2

<400>  13
aagaggttcc tgggtagtct tgagt                                             25


<210>  14
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 8.3

<400>  14
acgtacgttt aacaggatct cgggc                                             25


<210>  15
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PMO 8.4

<400>  15
cttcccttcc ctcaggtgaa tgaca                                             25


<210>  16
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  fRT primer

<400>  16
gtgatgaggt agaggaagag g                                                 21


<210>  17
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  rRT primer

<400>  17
gttgtagagc agggagagag                                                   20


<210>  18
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  fNes primer

<400>  18
cacagagaga accaccagca                                                   20


<210>  19
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  rNes primer

<400>  19
cttgacgttc tgcctcttcc                                                   20


<210>  20
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Control PMO

<400>  20
ccagcccatc ttctcctggt cctgg                                             25


<210>  21
<211>  2145
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  novel APP mRNA

<400>  21
atgctgcccg gtttggcact gctcctgctg gccgcctgga cggctcgggc gctggaggta       60

cccactgatg gtaatgctgg cctgctggct gaaccccaga ttgccatgtt ctgtggcaga      120

ctgaacatgc acatgaatgt ccagaatggg aagtgggatt cagatccatc agggaccaaa      180

acctgcattg ataccaagga aggcatcctg cagtattgcc aagaagtcta ccctgaactg      240

cagatcacca atgtggtaga agccaaccaa ccagtgacca tccagaactg gtgcaagcgg      300

ggccgcaagc agtgcaagac ccatccccac tttgtgattc cctaccgctg cttagttggt      360

gagtttgtaa gtgatgccct tctcgttcct gacaagtgca aattcttaca ccaggagagg      420

atggatgttt gcgaaactca tcttcactgg cacaccgtcg ccaaagagac atgcagtgag      480

aagagtacca acttgcatga ctacggcatg ttgctgccct gcggaattga caagttccga      540

ggggtagagt ttgtgtgttg cccactggct gaagaaagtg acaatgtgga ttctgctgat      600

gcggaggagg atgactcgga tgtctggtgg ggcggagcag acacagacta tgcagatggg      660

agtgaagaca aagtagtaga agtagcagag gaggaagaag tggctgaggt ggaagaagaa      720

gaagccgatg atgacgagga cgatgaggat ggtgatgagg tagaggaaga ggctgaggaa      780

ccctacgaag aagccacaga gagaaccacc agcattgcca ccaccaccac caccaccaca      840

gagtctgtgg aagaggtggt tcgagtgtcc caaagtttac tcaagactac ccaggaacct      900

cttgcccgag atcctgttaa acttcctaca acagcagcca gtacccctga tgccgttgac      960

aagtatctcg agacacctgg ggatgagaat gaacatgccc atttccagaa agccaaagag     1020

aggcttgagg ccaagcaccg agagagaatg tcccaggtca tgagagaatg ggaagaggca     1080

gaacgtcaag caaagaactt gcctaaagct gataagaagg cagttatcca gcatttccag     1140

gagaaagtgg aatctttgga acaggaagca gccaacgaga gacagcagct ggtggagaca     1200

cacatggcca gagtggaagc catgctcaat gaccgccgcc gcctggccct ggagaactac     1260

atcaccgctc tgcaggctgt tcctcctcgg cctcgtcacg tgttcaatat gctaaagaag     1320

tatgtccgcg cagaacagaa ggacagacag cacaccctaa agcatttcga gcatgtgcgc     1380

atggtggatc ccaagaaagc cgctcagatc cggtcccagg ttatgacaca cctccgtgtg     1440

atttatgagc gcatgaatca gtctctctcc ctgctctaca acgtgcctgc agtggccgag     1500

gagattcagg atgaagttga tgagctgctt cagaaagagc aaaactattc agatgacgtc     1560

ttggccaaca tgattagtga accaaggatc agttacggaa acgatgctct catgccatct     1620

ttgaccgaaa cgaaaaccac cgtggagctc cttcccgtga atggagagtt cagcctggac     1680

gatctccagc cgtggcattc ttttggggct gactctgtgc cagccaacac agaaaacgaa     1740

gttgagcctg ttgatgcccg ccctgctgcc gaccgaggac tgaccactcg accaggttct     1800

gggttgacaa atatcaagac ggaggagatc tctgaagtga agatggatgc agaattccga     1860

catgactcag gatatgaagt tcatcatcaa aaattggtgt tctttgcaga agatgtgggt     1920

tcaaacaaag gtgcaatcat tggactcatg gtgggcggtg ttgtcatagc gacagtgatc     1980

gtcatcacct tggtgatgct gaagaagaaa cagtacacat ccattcatca tggtgtggtg     2040

gaggttgacg ccgctgtcac cccagaggag cgccacctgt ccaagatgca gcagaacggc     2100

tacgaaaatc caacctacaa gttctttgag cagatgcaga actag                     2145


<210>  22
<211>  714
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Protein encoded by novel APP mRNA

<400>  22

Met Leu Pro Gly Leu Ala Leu Leu Leu Leu Ala Ala Trp Thr Ala Arg 
1               5                   10                  15      


Ala Leu Glu Val Pro Thr Asp Gly Asn Ala Gly Leu Leu Ala Glu Pro 
            20                  25                  30          


Gln Ile Ala Met Phe Cys Gly Arg Leu Asn Met His Met Asn Val Gln 
        35                  40                  45              


Asn Gly Lys Trp Asp Ser Asp Pro Ser Gly Thr Lys Thr Cys Ile Asp 
    50                  55                  60                  


Thr Lys Glu Gly Ile Leu Gln Tyr Cys Gln Glu Val Tyr Pro Glu Leu 
65                  70                  75                  80  


Gln Ile Thr Asn Val Val Glu Ala Asn Gln Pro Val Thr Ile Gln Asn 
                85                  90                  95      


Trp Cys Lys Arg Gly Arg Lys Gln Cys Lys Thr His Pro His Phe Val 
            100                 105                 110         


Ile Pro Tyr Arg Cys Leu Val Gly Glu Phe Val Ser Asp Ala Leu Leu 
        115                 120                 125             


Val Pro Asp Lys Cys Lys Phe Leu His Gln Glu Arg Met Asp Val Cys 
    130                 135                 140                 


Glu Thr His Leu His Trp His Thr Val Ala Lys Glu Thr Cys Ser Glu 
145                 150                 155                 160 


Lys Ser Thr Asn Leu His Asp Tyr Gly Met Leu Leu Pro Cys Gly Ile 
                165                 170                 175     


Asp Lys Phe Arg Gly Val Glu Phe Val Cys Cys Pro Leu Ala Glu Glu 
            180                 185                 190         


Ser Asp Asn Val Asp Ser Ala Asp Ala Glu Glu Asp Asp Ser Asp Val 
        195                 200                 205             


Trp Trp Gly Gly Ala Asp Thr Asp Tyr Ala Asp Gly Ser Glu Asp Lys 
    210                 215                 220                 


Val Val Glu Val Ala Glu Glu Glu Glu Val Ala Glu Val Glu Glu Glu 
225                 230                 235                 240 


Glu Ala Asp Asp Asp Glu Asp Asp Glu Asp Gly Asp Glu Val Glu Glu 
                245                 250                 255     


Glu Ala Glu Glu Pro Tyr Glu Glu Ala Thr Glu Arg Thr Thr Ser Ile 
            260                 265                 270         


Ala Thr Thr Thr Thr Thr Thr Thr Glu Ser Val Glu Glu Val Val Arg 
        275                 280                 285             


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


Pro Val Lys Leu Pro Thr Thr Ala Ala Ser Thr Pro Asp Ala Val Asp 
305                 310                 315                 320 


Lys Tyr Leu Glu Thr Pro Gly Asp Glu Asn Glu His Ala His Phe Gln 
                325                 330                 335     


Lys Ala Lys Glu Arg Leu Glu Ala Lys His Arg Glu Arg Met Ser Gln 
            340                 345                 350         


Val Met Arg Glu Trp Glu Glu Ala Glu Arg Gln Ala Lys Asn Leu Pro 
        355                 360                 365             


Lys Ala Asp Lys Lys Ala Val Ile Gln His Phe Gln Glu Lys Val Glu 
    370                 375                 380                 


Ser Leu Glu Gln Glu Ala Ala Asn Glu Arg Gln Gln Leu Val Glu Thr 
385                 390                 395                 400 


His Met Ala Arg Val Glu Ala Met Leu Asn Asp Arg Arg Arg Leu Ala 
                405                 410                 415     


Leu Glu Asn Tyr Ile Thr Ala Leu Gln Ala Val Pro Pro Arg Pro Arg 
            420                 425                 430         


His Val Phe Asn Met Leu Lys Lys Tyr Val Arg Ala Glu Gln Lys Asp 
        435                 440                 445             


Arg Gln His Thr Leu Lys His Phe Glu His Val Arg Met Val Asp Pro 
    450                 455                 460                 


Lys Lys Ala Ala Gln Ile Arg Ser Gln Val Met Thr His Leu Arg Val 
465                 470                 475                 480 


Ile Tyr Glu Arg Met Asn Gln Ser Leu Ser Leu Leu Tyr Asn Val Pro 
                485                 490                 495     


Ala Val Ala Glu Glu Ile Gln Asp Glu Val Asp Glu Leu Leu Gln Lys 
            500                 505                 510         


Glu Gln Asn Tyr Ser Asp Asp Val Leu Ala Asn Met Ile Ser Glu Pro 
        515                 520                 525             


Arg Ile Ser Tyr Gly Asn Asp Ala Leu Met Pro Ser Leu Thr Glu Thr 
    530                 535                 540                 


Lys Thr Thr Val Glu Leu Leu Pro Val Asn Gly Glu Phe Ser Leu Asp 
545                 550                 555                 560 


Asp Leu Gln Pro Trp His Ser Phe Gly Ala Asp Ser Val Pro Ala Asn 
                565                 570                 575     


Thr Glu Asn Glu Val Glu Pro Val Asp Ala Arg Pro Ala Ala Asp Arg 
            580                 585                 590         


Gly Leu Thr Thr Arg Pro Gly Ser Gly Leu Thr Asn Ile Lys Thr Glu 
        595                 600                 605             


Glu Ile Ser Glu Val Lys Met Asp Ala Glu Phe Arg His Asp Ser Gly 
    610                 615                 620                 


Tyr Glu Val His His Gln Lys Leu Val Phe Phe Ala Glu Asp Val Gly 
625                 630                 635                 640 


Ser Asn Lys Gly Ala Ile Ile Gly Leu Met Val Gly Gly Val Val Ile 
                645                 650                 655     


Ala Thr Val Ile Val Ile Thr Leu Val Met Leu Lys Lys Lys Gln Tyr 
            660                 665                 670         


Thr Ser Ile His His Gly Val Val Glu Val Asp Ala Ala Val Thr Pro 
        675                 680                 685             


Glu Glu Arg His Leu Ser Lys Met Gln Gln Asn Gly Tyr Glu Asn Pro 
    690                 695                 700                 


Thr Tyr Lys Phe Phe Glu Gln Met Gln Asn 
705                 710                 


<210>  23
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NEP f primer

<400>  23
cctggagatt cataatggat cttgt                                             25


<210>  24
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NEP R primer

<400>  24
aaagggcctt gcggaaag                                                     18


<210>  25
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  APP F primer

<400>  25
gatccatcag ggaccaaaac                                                   20


<210>  26
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  APP R primer

<400>  26
agcggtaggg aatcacaaag                                                   20


<210>  27
<211>  468
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target region of AON

<400>  27
taaattcctc agtaaatgtt tggtagatgc tgcctaataa accagtccag gttgccactg       60

ggaggattaa aagaagtaaa cgtgtataca tgaacagaga gacagtgcct tttcatgcta      120

aatgtggttc cccacatctc ctctgattag aggtgtgctc tgaacaagcc gagacggggc      180

cgtgccgagc aatgatctcc cgctggtact ttgatgtgac tgaagggaag tgtgccccat      240

tcttttacgg cggatgtggc ggcaaccgga acaactttga cacagaagag tactgcatgg      300

ccgtgtgtgg cagcgccagt aagtggaccc ttcttcgagc ctggccacct ttcgtctctc      360

tcgccactga ctctgctttt tgtaacagat tgattttcct ggttcttggg aatgggcctg      420

ttgctaccac taaccacatt tctgtccact tctctaattg ctcagagt                   468


<210>  28
<211>  357
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target region of AON

<400>  28
atgttcattt tggttttgtt ggagggacca aacctaagtg agtgattttg tttgttaggt       60

tgtttttttg tcagtggact cgtgcatttc agccatcatt cccatgtttc tctttttgtt      120

tttagttatg ttctcttatt ttttccatag tgtcccaaag tttactcaag actacccagg      180

aacctcttgc ccgagatcct gttaaacgta cgttgtcatt cacctgaggg aagggaagag      240

gggaggagga tgctgcttgg ttcacataac tccagcatca tcaccttctt tgcatggttt      300

tgtgtttctt gaacacctgt cttagtaaaa tgtttcttcc cattaccttg cttgtaa         357


