                         SEQUENCE LISTING

<110>  ProQR Therapeutics B.V.
 
<120>  Oligonucleotides for making a change in the sequence of a target 
       RNA  molecule present in a living cell

<130>  P6042219PCT

<160>  70    

<170>  PatentIn version 3.3

<210>  1
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide

<400>  1
aucauaggaa acaccaaaga ugauauuuuc uuu                                  33


<210>  2
<211>  11
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  U is phosphorylated

<220>
<221>  misc_feature
<222>  (11)..(11)
<223>  U is phosphorylated

<400>  2
ucauuuuugg u                                                          11


<210>  3
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide

<400>  3
aucauaggaa acaccaaaaa ugauauuuuc uuu                                  33


<210>  4
<211>  11
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  U is phosphorylated

<220>
<221>  misc_feature
<222>  (11)..(11)
<223>  U is phosphorylated

<400>  4
ucaucuuugg u                                                          11


<210>  5
<211>  63
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10  of the CFTR transcript

<400>  5
uaugccuggc accauuaaag aaaauaucau cuuugguguu uccuaugaug aauauagaua     60

cag                                                                   63


<210>  6
<211>  60
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  6
uaugccuggc accauuaaag aaaauaucau ugguguuucc uaugaugaau auagauacag     60


<210>  7
<211>  30
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  7
uaugccuggc accauuaaag aaaauaucau                                      30


<210>  8
<211>  30
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  8
ugguguuucc uaugaugaau auagauacag                                      30


<210>  9
<211>  63
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR trancript

<400>  9
uaugccuggc accauuaaag aaaauaucau cuuugguguu uccuaugaug aauauagaua     60

cag                                                                   63


<210>  10
<211>  60
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  10
augccuggca ccauuaaaga aaauaucauc uuugguguuu ccuaugauga auauagauac     60


<210>  11
<211>  20
<212>  PRT
<213>  Artificial

<220>
<223>  Peptide

<400>  11

Met Pro Gly Thr Ile Lys Glu Asn Ile Ile Phe Gly Val Ser Tyr Asp 
1               5                   10                  15      


Glu Tyr Arg Tyr 
            20  


<210>  12
<211>  57
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  12
augccuggca ccauuaaaga aaauaucauu gguguuuccu augaugaaua uagauac        57


<210>  13
<211>  19
<212>  PRT
<213>  Artificial

<220>
<223>  Peptide

<400>  13

Met Pro Gly Thr Ile Lys Glu Asn Ile Ile Gly Val Ser Tyr Asp Glu 
1               5                   10                  15      


Tyr Arg Tyr 
            


<210>  14
<211>  60
<212>  RNA
<213>  Artificial

<220>
<223>  Part of exon 10 of the CFTR transcript

<400>  14
augccuggca ccauuaaaga aaauaucauc uuugguguuu ccuaugauga auauagauac     60


<210>  15
<211>  20
<212>  PRT
<213>  Artificial

<220>
<223>  Peptide

<400>  15

Met Pro Gly Thr Ile Lys Glu Asn Ile Ile Phe Gly Val Ser Tyr Asp 
1               5                   10                  15      


Glu Tyr Arg Tyr 
            20  


<210>  16
<211>  32
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide dF-ins1

<400>  16
aucauaggaa acaccaagau gauauuuucu uu                                   32


<210>  17
<211>  31
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide dF-ins1

<400>  17
aucauaggaa acaccagaug auauuuucuu u                                    31


<210>  18
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide dF-ins1 33b

<400>  18
caucauagga aacaccagau gauauuuucu uua                                  33


<210>  19
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT X

<400>  19
auaggaaaca ccaaaucaga ugauauuuuc uuu                                  33


<210>  20
<211>  32
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT Ins2

<400>  20
auaggaaaca ccaaaucgau gauauuuucu uu                                   32


<210>  21
<211>  31
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT Ins1

<400>  21
auaggaaaca ccaaaugaug auauuuucuu u                                    31


<210>  22
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT L

<400>  22
auaggaaaca ccaaagagga ugauauuuuc uuu                                  33


<210>  23
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT OX

<400>  23
aggcauaauc caggaucaaa acugagaaca gaa                                  33


<210>  24
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR WT OL

<400>  24
auggugccag gcauaaggau ccaggaaaac uga                                  33


<210>  25
<211>  42
<212>  RNA
<213>  Artificial

<220>
<223>  Part of deltaF508 RNA

<400>  25
accauuaaag aaaauaucau ugguguuucc uaugaugaau au                        42


<210>  26
<211>  14
<212>  PRT
<213>  Artificial

<220>
<223>  Part of delta F508 polypeptide

<400>  26

Thr Ile Lys Glu Asn Ile Ile Gly Val Ser Tyr Asp Glu Tyr 
1               5                   10                  


<210>  27
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of WT RNA

<400>  27
accauuaaag aaaauaucau cuuugguguu uccuaugaug aauau                     45


<210>  28
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of WT polypeptide

<400>  28

Thr Ile Lys Glu Asn Ile Ile Phe Gly Val Ser Tyr Asp Glu Tyr 
1               5                   10                  15  


<210>  29
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of delta F508 RNA with CU insertion

<400>  29
accauuaaag aaaauaucau cuugguguuu ccuaugauga auaua                     45


<210>  30
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of delta 508 polypeptide, with frameshift

<400>  30

Thr Ile Lys Glu Asn Ile Ile Leu Val Phe Pro Met Met Asn Ile 
1               5                   10                  15  


<210>  31
<211>  43
<212>  RNA
<213>  Artificial

<220>
<223>  Part of delta F508 RNA, with C insertion

<400>  31
accauuaaag aaaauaucau cugguguuuc cuaugaugaa uau                       43


<210>  32
<211>  11
<212>  PRT
<213>  Artificial

<220>
<223>  Part of delta F508 polypeptide with premature stop

<400>  32

Thr Ile Lys Glu Asn Ile Ile Trp Cys Phe Leu 
1               5                   10      


<210>  33
<211>  18
<212>  DNA
<213>  Artificial

<220>
<223>  Part of delta F508 DNA with stopcodon at position 508

<400>  33
atcatctgat ttggtgtt                                                   18


<210>  34
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of delta F 508 RNA, with stopcodon at position 508

<400>  34
accauuaaag aaaauaucau cugauuuggu guuuccuaug augaa                     45


<210>  35
<211>  7
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide with stopcodon inserted at position 
       508

<400>  35

Thr Ile Lys Glu Asn Ile Ile 
1               5           


<210>  36
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA with GA insertion

<400>  36
accauuaaag aaaauaucau cgauuuggug uuuccuauga ugaau                     45


<210>  37
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide with frameshift induced by GA 
       insertion

<400>  37

Thr Ile Lys Glu Asn Ile Ile Asp Leu Val Phe Pro Met Met Asn 
1               5                   10                  15  


<210>  38
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA with A insertion

<400>  38
accauuaaag aaaauaucau cauuuggugu uuccuaugau gaaua                     45


<210>  39
<211>  12
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide with frameshift induced stopcodon at 
       position 513

<400>  39

Thr Ile Lys Glu Asn Ile Ile Ile Trp Cys Phe Leu 
1               5                   10          


<210>  40
<211>  18
<212>  DNA
<213>  Artificial

<220>
<223>  Part of CFTR DNA with Leucine codon inserted at codon position 
       508

<400>  40
atcatcctct ttggtgtt                                                   18


<210>  41
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of the CFTR WT RNA with Leucine codon (CUC) insertion at 
       position 508

<400>  41
accauuaaag aaaauaucau ccucuuuggu guuuccuaug augaa                     45


<210>  42
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide with Leucine inserted at position 508

<400>  42

Thr Ile Lys Glu Asn Ile Ile Leu Phe Gly Val Ser Tyr Asp Glu 
1               5                   10                  15  


<210>  43
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  43
auuucauucu guucucaguu uuccuggauu augccuggca ccauu                     45


<210>  44
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part CFTR WT polypeptide

<400>  44

Ile Ser Phe Cys Ser Gln Phe Ser Trp Ile Met Pro Gly Thr Ile 
1               5                   10                  15  


<210>  45
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA with stopcodon insertion at codon position 
       495

<400>  45
auuucauucu guucucaguu uugauccugg auuaugccug gcacc                     45


<210>  46
<211>  7
<212>  PRT
<213>  Artificial

<220>
<223>  Part of truncated CFTR WT polypeptide due to stopcodon inserted 
       at position 495

<400>  46

Ile Ser Phe Cys Ser Gln Phe 
1               5           


<210>  47
<211>  48
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  47
uucuguucuc aguuuuccug gauuaugccu ggcaccauua aagaaaau                  48


<210>  48
<211>  16
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide

<400>  48

Phe Cys Ser Gln Phe Ser Trp Ile Met Pro Gly Thr Ile Lys Glu Asn 
1               5                   10                  15      


<210>  49
<211>  48
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA with Leucine codon insertion at position 498

<400>  49
uucuguucuc aguuuuccug gauccuuaug ccuggcacca uuaaagaa                  48


<210>  50
<211>  16
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide with Leucine insertion at position 
       498

<400>  50

Phe Cys Ser Gln Phe Ser Trp Ile Leu Met Pro Gly Thr Ile Lys Glu 
1               5                   10                  15      


<210>  51
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  Oligonucleotide CFTR-R117

<400>  51
auaaaucgcg auagagcguu ccuccuuguu auc                                  33


<210>  52
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of 117H RNA

<400>  52
gacccggaua acaaggagga acacucuauc gcgauuuauc uaggc                     45


<210>  53
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of 117H polypeptide

<400>  53

Asp Pro Asp Asn Lys Glu Glu His Ser Ile Ala Ile Tyr Leu Gly 
1               5                   10                  15  


<210>  54
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  54
gacccggaua acaaggagga acgcucuauc gcgauuuauc uaggc                     45


<210>  55
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide

<400>  55

Asp Pro Asp Asn Lys Glu Glu Arg Ser Ile Ala Ile Tyr Leu Gly 
1               5                   10                  15  


<210>  56
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  oligonucleotide CFTR-G542

<400>  56
gugauuccac cuucuccaag aacuauauug ucu                                  33


<210>  57
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of 542X RNA

<400>  57
gagaaagaca auauaguucu uugagaaggu ggaaucacac ugagu                     45


<210>  58
<211>  7
<212>  PRT
<213>  Artificial

<220>
<223>  Part of 542X truncated polypeptide

<400>  58

Glu Lys Asp Asn Ile Val Leu 
1               5           


<210>  59
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  59
gagaaagaca auauaguucu uggagaaggu ggaaucacac ugagu                     45


<210>  60
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide

<400>  60

Glu Lys Asp Asn Ile Val Leu Gly Glu Gly Gly Ile Thr Leu Ser 
1               5                   10                  15  


<210>  61
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  oligonucleotide CFTR-W1282

<400>  61
cuccaaaggc uuuccuccac uguugcaaag uua                                  33


<210>  62
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of 1282X RNA

<400>  62
gauucaauaa cuuugcaaca gugaaggaaa gccuuuggag ugaua                     45


<210>  63
<211>  7
<212>  PRT
<213>  Artificial

<220>
<223>  Part of 1282X truncated polypeptide

<400>  63

Asp Ser Ile Thr Leu Gln Gln 
1               5           


<210>  64
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  64
gauucaauaa cuuugcaaca guggaggaaa gccuuuggag ugaua                     45


<210>  65
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide

<400>  65

Asp Ser Ile Thr Leu Gln Gln Trp Arg Lys Ala Phe Gly Val Ile 
1               5                   10                  15  


<210>  66
<211>  33
<212>  RNA
<213>  Artificial

<220>
<223>  oligonucleotide CFTR-N1303

<400>  66
guucauaggg auccaaguuu uuucuaaaug uuc                                  33


<210>  67
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of 1303K RNA

<400>  67
uuuucuggaa cauuuagaaa aaaguuggau cccuaugaac agugg                     45


<210>  68
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of 1303K PP

<400>  68

Phe Ser Gly Thr Phe Arg Lys Lys Leu Asp Pro Tyr Glu Gln Trp 
1               5                   10                  15  


<210>  69
<211>  45
<212>  RNA
<213>  Artificial

<220>
<223>  Part of CFTR WT RNA

<400>  69
uuuucuggaa cauuuagaaa aaacuuggau cccuaugaac agugg                     45


<210>  70
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Part of CFTR WT polypeptide

<400>  70

Phe Ser Gly Thr Phe Arg Lys Asn Leu Asp Pro Tyr Glu Gln Trp 
1               5                   10                  15  

