                         SEQUENCE LISTING

<110>  uniQure IP B.V.
 
<120>  A COMPANION DIAGNOSIC TO MONITOR THE EFFECTS OF GENE THERAPY

<130>  402692_2_WO

<140>  PCT/EP2019/081759
<141>  2019-11-19

<160>  24    

<170>  PatentIn version 3.5

<210>  1
<211>  2440
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miR451 scaffold expression construct

<400>  1
cagtcacgac gttgtaaaac gacggccagt gaattcgccc ttcaattcgg tacctagtta       60

ttaatagtaa tcaattacgg ggtcattagt tcatagccca tatatggagt tccgcgttac      120

ataacttacg gtaaatggcc cgcctggctg accgcccaac gacccccgcc cattgacgtc      180

aataatgacg tatgttccca tagtaacgcc aatagggact ttccattgac gtcaatgggt      240

ggagtattta cggtaaactg cccacttggc agtacatcaa gtgtatcata tgccaagtac      300

gccccctatt gacgtcaatg acggtaaatg gcccgcctgg cattatgccc agtacatgac      360

cttatgggac tttcctactt ggcagtacat ctacgtatta gtcatcgcta ttaccatggt      420

cgaggtgagc cccacgttct gcttcactct ccccatctcc cccccctccc cacccccaat      480

tttgtattta tttatttttt aattattttg tgcagcgatg ggggcggggg gggggggggg      540

gcgcgcgcca ggcggggcgg ggcggggcga ggggcggggc ggggcgaggc ggagaggtgc      600

ggcggcagcc aatcagagcg gcgcgctccg aaagtttcct tttatggcga ggcggcggcg      660

gcggcggccc tataaaaagc gaagcgcgcg gcgggcggga gtcgctgcgc gctgccttcg      720

ccccgtgccc cgctccgccg ccgcctcgcg ccgcccgccc cggctctgac tgaccgcgtt      780

actcccacag gtgagcgggc gggacggccc ttctcctccg ggctgtaatt agcgcttggt      840

ttaatgacgg cttgtttctt ttctgtggct gcgtgaaagc cttgaggggc tccgggaggg      900

ccctttgtgc ggggggagcg gctcgggggg tgcgtgcgtg tgtgtgtgcg tggggagcgc      960

cgcgtgcggc tccgcgctgc ccggcggctg tgagcgctgc gggcgcggcg cggggctttg     1020

tgcgctccgc agtgtgcgcg aggggagcgc ggccgggggc ggtgccccgc ggtgcggggg     1080

gggctgcgag gggaacaaag gctgcgtgcg gggtgtgtgc gtgggggggt gagcaggggg     1140

tgtgggcgcg tcggtcgggc tgcaaccccc cctgcacccc cctccccgag ttgctgagca     1200

cggcccggct tcgggtgcgg ggctccgtac ggggcgtggc gcggggctcg ccgtgccggg     1260

cggggggtgg cggcaggtgg gggtgccggg cggggcgggg ccgcctcggg ccggggaggg     1320

ctcgggggag gggcgcggcg gcccccggag cgccggcggc tgtcgaggcg cggcgagccg     1380

cagccattgc ttttatggta atcgtgcgag agggcgcagg gacttccttt gtcccaaatc     1440

tgtgcggagc cgaaatctgg gaggcgccgc cgcaccccct ctagcgggcg cggggcgaag     1500

cggtgcggcg ccggcaggaa ggaaatgggc ggggagggcc ttcgtgcgtc gccgcgccgc     1560

cgtccccttc tccctctcca gcctcggggc tgtccgcggg gggacggctg ccttcggggg     1620

ggacggggca gggcggggtt cggcttctgg cgtgtgaccg gcggctctag agcctctgct     1680

aaccatgttc atgccttctt ctttttccta cagctcctgg gcaacgtgct ggttattgtg     1740

ctgtctcatc attttggcaa agaattaagg gcgaattcga gctcggtacc tcgcgaatgc     1800

atctagatat cggcgctatg cttcctgtgc ccccagtggg gccctggctg ggatttcatc     1860

atatactgta agtttgcgat gagacactac agtatagatg atgtactagt ccgggcaccc     1920

ccagctctgg agcctgacaa ggaggacagg agagatgctg caagcccaag aagctctctg     1980

ctcagcctgt cacaacctac tgactgccag ggcacttggg aatggcaagg tctggaacta     2040

ccttgcatac ttatgcaagg tagttccagt cttgctatac ccagaaaacg tgccaggaag     2100

agaactcagg accctgaagc agactactgg aagggagact ccagctcaaa caaggcaggg     2160

gtgggggcgt gggattgggg gtaggggagg gaatagatac attttctctt tcctgttgta     2220

aagaaataaa gataagccag gcacagtggc tcacgcctgt aatcccacca ctttcagagg     2280

ccaaggcgct ggatccagat ctcgagcggc cgcccgtggc atccctgtga cccctcccca     2340

gtgcctctcc tggccctgga agttgccact ccagtgccca ccagccttgt cctaataaaa     2400

ttaagttgca tcaagatcga cgggcccgtc gactgcagag                           2440


<210>  2
<211>  22
<212>  DNA
<213>  Homo sapiens ATXN3

<400>  2
tctggaacta ccttgcatac tt                                                22


<210>  3
<211>  22
<212>  DNA
<213>  Homo sapiens - HTT

<400>  3
aaggacttga gggactcgaa ga                                                22


<210>  4
<211>  72
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  HTT miRNA encoding sequence

<400>  4
cttgggaatg gcaaggaagg acttgaggga ctcgaagacg agtccctcaa gtcctctctt       60

gctataccca ga                                                           72


<210>  5
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  RNA sequences complementary to HTT

<400>  5
aaggacuuga gggacucgaa ga                                                22


<210>  6
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  RNA sequences complementary to ATNX3

<400>  6
ucuggaacua ccuugcauac uu                                                22


<210>  7
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  7
aaggacttga gggactcgaa gacg                                              24


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

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  8
aaggacttga gggactcgaa gacgagtccc                                        30


<210>  9
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  9
aaggacttga gggactcgaa gac                                               23


<210>  10
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  10
aaggacttga gggactcgaa gacga                                             25


<210>  11
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  11
aaggacttga gggactcgaa g                                                 21


<210>  12
<211>  27
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  12
aaggacttga gggactcgaa gacgagt                                           27


<210>  13
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  13
aaggacttga gggactcga                                                    19


<210>  14
<211>  31
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  14
aaggacttga gggactcgaa gacgagtccc t                                      31


<210>  15
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of sequences produced from the scaffold 
       targeting Huntington

<400>  15
aaggacttga gggactcgaa                                                   20


<210>  16
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of tsequences produced from the scaffold 
       targeting ATXN3

<400>  16
tctggaacta ccttgcatac ttat                                              24


<210>  17
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of tsequences produced from the scaffold 
       targeting ATXN3

<400>  17
tctggaacta ccttgcatac tta                                               23


<210>  18
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of tsequences produced from the scaffold 
       targeting ATXN3

<400>  18
tctggaacta ccttgcatac t                                                 21


<210>  19
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Small RNAseq analysis of tsequences produced from the scaffold 
       targeting ATXN3

<400>  19
tctggaacta ccttgcatac ttatgcaagg                                        30


<210>  20
<211>  16
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  miRNA451 scaffold 5'sequence

<400>  20
cuugggaaug gcaagg                                                       16


<210>  21
<211>  17
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  miRNA451 scaffold 3'sequience

<400>  21
cucuugcuau acccaga                                                      17


<210>  22
<211>  71
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Pre-miC_451


<220>
<221>  misc_feature
<222>  (17)..(55)
<223>  n is a, c, g, or u

<400>  22
cuugggaaug gcaaggnnnn nnnnnnnnnn nnnnnnnnnn nnnnnnnnnn nnnnnucuug       60

cuauacccag a                                                            71


<210>  23
<211>  16
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  forward primer sequence

<400>  23
gagccgcagc cattgc                                                       16


<210>  24
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  reverse primer sequence

<400>  24
cacagatttg ggacaaagga agt                                               23


