                         SEQUENCE LISTING

<110>  KORRO BIO, INC.
 
<120>  METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF ABCA4

<130>  01249-0003-00PCT

<150>  US 62/704,568
<151>  2020-05-15

<160>  90    

<170>  PatentIn version 3.5

<210>  1
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  1
ggugaauagu auaacaauau                                                   20


<210>  2
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  2
auguuguuau aguauccacc                                                   20


<210>  3
<211>  45
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  3
ggugaauagu auaacaauau gcuaaauguu guuauaguau ccacc                       45


<210>  4
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  4
ggugaagagg agaacaauau                                                   20


<210>  5
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  5
auguuguucu cgucuccacc                                                   20


<210>  6
<211>  45
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  6
ggugaagagg agaacaauau gcuaaauguu guucucgucu ccacc                       45


<210>  7
<211>  25
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  7
ggugucgaga agaggagaac aauau                                             25


<210>  8
<211>  25
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  8
auguuguucu cgucuccucg acacc                                             25


<210>  9
<211>  55
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  9
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acacc            55


<210>  10
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  10
ggguggaaua guauaacaau au                                                22


<210>  11
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  11
auguuguuau aguaucccac cu                                                22


<210>  12
<211>  49
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  12
ggguggaaua guauaacaau augcuaaaug uuguuauagu aucccaccu                   49


<210>  13
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  13
guggaauagu auaacaauau                                                   20


<210>  14
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  14
auguuguuau aguaucccac                                                   20


<210>  15
<211>  45
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  15
guggaauagu auaacaauau gcuaaauguu guuauaguau cccac                       45


<210>  16
<211>  25
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  16
ggugucgaga auaguauaac aauau                                             25


<210>  17
<211>  25
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  17
auguuguuau aguauccucg acacc                                             25


<210>  18
<211>  55
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  18
ggugucgaga auaguauaac aauaugcuaa auguuguuau aguauccucg acacc            55


<210>  19
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  19
ggguggaaua guauaacaau au                                                22


<210>  20
<211>  22
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  20
auguuguuau aguaucccac cu                                                22


<210>  21
<211>  49
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  21
ggguggaaua guauaacaau augcuaaaug uuguuauagu aucccaccu                   49


<210>  22
<211>  18
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  22
ggguggaaua guauacca                                                     18


<210>  23
<211>  18
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  23
ugguauagua ucccaccu                                                     18


<210>  24
<211>  40
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  24
ggguggaaua guauaccauu cgugguauag uaucccaccu                             40


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

<220>
<223>  Synthetic: Nucleobase sequence

<400>  25
guggguggaa uaguauacca                                                   20


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

<220>
<223>  Synthetic: Nucleobase sequence

<400>  26
ugguauagua ucccaccuac                                                   20


<210>  27
<211>  44
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  27
guggguggaa uaguauacca uucgugguau aguaucccac cuac                        44


<210>  28
<211>  19
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  28
uggguggaau aguauacca                                                    19


<210>  29
<211>  19
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  29
ugguauagua ucccaccua                                                    19


<210>  30
<211>  42
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  30
uggguggaau aguauaccau ucgugguaua guaucccacc ua                          42


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

<220>
<223>  Synthetic: Nucleobase sequence

<400>  31
gguggaauag uauacca                                                      17


<210>  32
<211>  17
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  32
ugguauagua ucccacc                                                      17


<210>  33
<211>  38
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  33
gguggaauag uauaccauuc gugguauagu aucccacc                               38


<210>  34
<211>  16
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  34
guggaauagu auacca                                                       16


<210>  35
<211>  16
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Nucleobase sequence

<400>  35
ugguauagua ucccac                                                       16


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

<220>
<223>  Synthetic: Nucleobase sequence

<400>  36
guggaauagu auaccauucg ugguauagua ucccac                                 36


<210>  37
<211>  45
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  37
ggugaauagu auaacaauau gcuaaauguu guuauaguau ccacc                       45


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

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  38
ggugaagagg agaacaauau gcuaaauguu guucucgucu ccacc                       45


<210>  39
<211>  55
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  39
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acacc            55


<210>  40
<211>  27
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  40
ggagaagagg agaaaaagaa aggggaa                                           27


<210>  41
<211>  49
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  41
ggguggaaua guauaacaau augcuaaaug uuguuauagu aucccaccu                   49


<210>  42
<211>  45
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  42
guggaauagu auaacaauau gcuaaauguu guuauaguau cccac                       45


<210>  43
<211>  55
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  43
ggugucgaga auaguauaac aauaugcuaa auguuguuau aguauccucg acacc            55


<210>  44
<211>  49
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  44
ggguggaaua guauaacaau augcuaaaug uuguuauagu aucccaccu                   49


<210>  45
<211>  40
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  45
ggguggaaua guauaccauu cgugguauag uaucccaccu                             40


<210>  46
<211>  44
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  46
guggguggaa uaguauacca uucgugguau aguaucccac cuac                        44


<210>  47
<211>  42
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  47
uggguggaau aguauaccau ucgugguaua guaucccacc ua                          42


<210>  48
<211>  38
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  48
gguggaauag uauaccauuc gugguauagu aucccacc                               38


<210>  49
<211>  36
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: GluR2 ADAR-recruiting domain


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of preferred 
       embodiments and modifications

<400>  49
guggaauagu auaccauucg ugguauagua ucccac                                 36


<210>  50
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: MS2 ADAR-recruiting domain

<400>  50
acatgaggat cacccatgt                                                    19


<210>  51
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Primer - ABCA4 fwd

<400>  51
gtggtgtact tcctcctgac                                                   20


<210>  52
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Primer - ABCA4 rvs

<400>  52
gcataaaggt aaagatgttc tcgtc                                             25


<210>  53
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Fully modified with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  53
acucccagga ggccaaagca cucuccaggg cgaactccga cacacagccu                  50


<210>  54
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Fully modified with DNA triplet and 10-NT
       gap mismatches


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  54
acucccagga ggccaaagca gucuacaggg cgaactccga cacacagccu                  50


<210>  55
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Fully modified with DNA triplet and 15-NT
       gap mismatches


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  55
acucccagga ggccacagcc cucuccaggg cgaactccga cacacagccu                  50


<210>  56
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified dsRBD motif and fully 2'-OMe 
       modified specificity motif with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  56
acucccagga ggccaaagca cucuccaggg cgaactccga cacacagccu                  50


<210>  57
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified dsRBD motif and fully 2'-OMe 
       modified specificity motif with DNA triplet and mismatches with 
       10-NT gap


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  57
acucccagga ggccaaagca gucuacaggg cgaactccga cacacagccu                  50


<210>  58
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified dsRBD motif and fully 2'-OMe 
       modified specificity motif with DNA triplet and mismatches with 
       15-NT gap


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  58
acucccagga ggccacagcc cucuccaggg cgaactccga cacacagccu                  50


<210>  59
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Partially modified dsRBD motif (Pu: RNA &
       Py: 2'-OMe) and fully 2'-OMe modified specificity motif with DNA 
       triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  59
acucccagga ggccaaagca cucuccaggg cgaactccga cacacagccu                  50


<210>  60
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Partially 2'-OMe modified dsRBD based on 
       GluR and fully 2'-OMe modified specificity motif with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  60
acucccagga ggccaaagca cucuccaggg cgaactccga cacacagccu                  50


<210>  61
<211>  50
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Fully 2'-OMe modified with FANA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  61
acucccagga ggccaaagca cucuccaggg cgaacuccga cacacagccu                  50


<210>  62
<211>  50
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified dsRBD motif and fully 2'-OMe 
       modified specificity motif with FANA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  62
acucccagga ggccaaagca cucuccaggg cgaacuccga cacacagccu                  50


<210>  63
<211>  73
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Fully modified G/C-rich longer GluR 
       (55-mer)


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  63
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccgcgaa       60

ctccgacaca cag                                                          73


<210>  64
<211>  73
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified G/C-rich longer GluR (55-mer)


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  64
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccgcgaa       60

ctccgacaca cag                                                          73


<210>  65
<211>  73
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Partially modified G/C-rich longer GluR 
       and fully modified specificity motif with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  65
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccgcgaa       60

ctccgacaca cag                                                          73


<210>  66
<211>  95
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Unmodified G/C-rich longer GluR with 
       partially modified longer specificity motif with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  66
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccggagg       60

ccaaagcacu cuccagggcg aactccgaca cacag                                  95


<210>  67
<211>  95
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Partially modified G/C-rich longer GluR 
       and longer specificity motifs with DNA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  67
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccggagg       60

ccaaagcacu cuccagggcg aactccgaca cacag                                  95


<210>  68
<211>  95
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Synthetic: Guide RNA -  Partially modified G/C-rich longer GluR 
       and longer specity motif with FANA triplet


<220>
<221>  misc_feature
<223>  see specification as filed for detailed description of 
       modifications

<400>  68
ggugucgaga agaggagaac aauaugcuaa auguuguucu cgucuccucg acaccggagg       60

ccaaagcacu cuccagggcg aacuccgaca cacag                                  95


<210>  69

<400>  69
000

<210>  70

<400>  70
000

<210>  71

<400>  71
000

<210>  72

<400>  72
000

<210>  73

<400>  73
000

<210>  74

<400>  74
000

<210>  75

<400>  75
000

<210>  76

<400>  76
000

<210>  77

<400>  77
000

<210>  78

<400>  78
000

<210>  79

<400>  79
000

<210>  80

<400>  80
000

<210>  81

<400>  81
000

<210>  82

<400>  82
000

<210>  83

<400>  83
000

<210>  84

<400>  84
000

<210>  85

<400>  85
000

<210>  86

<400>  86
000

<210>  87
<211>  16
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic: exemplary hydrophobic MTS-containing peptide

<400>  87

Ala Ala Val Ala Leu Leu Pro Ala Val Leu Leu Ala Leu Leu Ala Pro 
1               5                   10                  15      


<210>  88
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic: RFGF analogue

<400>  88

Ala Ala Leu Leu Pro Val Leu Leu Ala Ala Pro 
1               5                   10      


<210>  89
<211>  13
<212>  PRT
<213>  Human immunodeficiency virus


<220>
<221>  misc_feature
<223>  HIV Tat protein

<400>  89

Gly Arg Lys Lys Arg Arg Gln Arg Arg Arg Pro Pro Gln 
1               5                   10              


<210>  90
<211>  16
<212>  PRT
<213>  Drosophila sp.


<220>
<221>  misc_feature
<223>  Drosophila Antennapedia protein

<400>  90

Arg Gln Ile Lys Ile Trp Phe Gln Asn Arg Arg Met Lys Trp Lys Lys 
1               5                   10                  15      


