                         SEQUENCE LISTING

<110>  St. Jude Children's Research Hospital, Inc.
 
<120>  SYSTEMS AND METHODS FOR BASE EDITING OF HBG1/2 GENE PROMOTER AND 
       FETAL HEMOGLOBIN INDUCTION

<130>  SJ-20-0020 (41901-7)

<140>  PCT/US2022/012497
<141>  2022-01-14

<150>  63/137,919
<151>  2021-01-15

<160>  28    

<170>  PatentIn version 3.5

<210>  1
<211>  107
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  sgRNA-198


<220>
<221>  misc_feature
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (2)..(2)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (4)..(4)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (6)..(6)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (102)..(102)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (104)..(104)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (106)..(106)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<400>  1
gngnungggg aaggggcccc caagguuuua gagcuagaaa uagcaaguua aaauaaggcu       60

aguccguuau caacuugaaa aaguggcacc gagucggugc unununu                    107


<210>  2
<211>  107
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  sgRNA-175


<220>
<221>  misc_feature
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (2)..(2)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (4)..(4)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (6)..(6)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (102)..(102)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (104)..(104)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (106)..(106)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<400>  2
gnangnauau uugcauugag auagguuuua gagcuagaaa uagcaaguua aaauaaggcu       60

aguccguuau caacuugaaa aaguggcacc gagucggugc unununu                    107


<210>  3
<211>  107
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  sgRNA-113


<220>
<221>  misc_feature
<222>  (2)..(2)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (4)..(4)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (6)..(6)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (102)..(102)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (104)..(104)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<220>
<221>  misc_feature
<222>  (106)..(106)
<223>  2'-O-methyl modification and phosphorothioate inter-nucleotide 
       linkage

<400>  3
gncnunugac caauagccuu gacaguuuua gagcuagaaa uagcaaguua aaauaaggcu       60

aguccguuau caacuugaaa aaguggcacc gagucggugc unununu                    107


<210>  4
<211>  5412
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ABE-max NG SpCas9 plasmid ORF

<400>  4
atgaaacgga cagccgacgg aagcgagttc gagtcaccaa agaagaagcg gaaagtctct       60

gaagtcgagt ttagccacga gtattggatg aggcacgcac tgaccctggc aaagcgagca      120

tgggatgaaa gagaagtccc cgtgggcgcc gtgctggtgc acaacaatag agtgatcgga      180

gagggatgga acaggccaat cggccgccac gaccctaccg cacacgcaga gatcatggca      240

ctgaggcagg gaggcctggt catgcagaat taccgcctga tcgatgccac cctgtatgtg      300

acactggagc catgcgtgat gtgcgcagga gcaatgatcc acagcaggat cggaagagtg      360

gtgttcggag cacgggacgc caagaccggc gcagcaggct ccctgatgga tgtgctgcac      420

caccccggca tgaaccaccg ggtggagatc acagagggaa tcctggcaga cgagtgcgcc      480

gccctgctga gcgatttctt tagaatgcgg agacaggaga tcaaggccca gaagaaggca      540

cagagctcca ccgactctgg aggatctagc ggaggatcct ctggaagcga gacaccaggc      600

acaagcgagt ccgccacacc agagagctcc ggcggctcct ccggaggatc ctctgaggtg      660

gagttttccc acgagtactg gatgagacat gccctgaccc tggccaagag ggcacgcgat      720

gagagggagg tgcctgtggg agccgtgctg gtgctgaaca atagagtgat cggcgagggc      780

tggaacagag ccatcggcct gcacgaccca acagcccatg ccgaaattat ggccctgaga      840

cagggcggcc tggtcatgca gaactacaga ctgattgacg ccaccctgta cgtgacattc      900

gagccttgcg tgatgtgcgc cggcgccatg atccactcta ggatcggccg cgtggtgttt      960

ggcgtgagga acgcaaaaac cggcgccgca ggctccctga tggacgtgct gcactacccc     1020

ggcatgaatc accgcgtcga aattaccgag ggaatcctgg cagatgaatg tgccgccctg     1080

ctgtgctatt tctttcggat gcctagacag gtgttcaatg ctcagaagaa ggcccagagc     1140

tccaccgact ccggaggatc tagcggaggc tcctctggct ctgagacacc tggcacaagc     1200

gagagcgcaa cacctgaaag cagcgggggc agcagcgggg ggtcagacaa gaagtacagc     1260

atcggcctgg ccatcggcac caactctgtg ggctgggccg tgatcaccga cgagtacaag     1320

gtgcccagca agaaattcaa ggtgctgggc aacaccgacc ggcacagcat caagaagaac     1380

ctgatcggag ccctgctgtt cgacagcggc gaaacagccg aggccacccg gctgaagaga     1440

accgccagaa gaagatacac cagacggaag aaccggatct gctatctgca agagatcttc     1500

agcaacgaga tggccaaggt ggacgacagc ttcttccaca gactggaaga gtccttcctg     1560

gtggaagagg ataagaagca cgagcggcac cccatcttcg gcaacatcgt ggacgaggtg     1620

gcctaccacg agaagtaccc caccatctac cacctgagaa agaaactggt ggacagcacc     1680

gacaaggccg acctgcggct gatctatctg gccctggccc acatgatcaa gttccggggc     1740

cacttcctga tcgagggcga cctgaacccc gacaacagcg acgtggacaa gctgttcatc     1800

cagctggtgc agacctacaa ccagctgttc gaggaaaacc ccatcaacgc cagcggcgtg     1860

gacgccaagg ccatcctgtc tgccagactg agcaagagca gacggctgga aaatctgatc     1920

gcccagctgc ccggcgagaa gaagaatggc ctgttcggaa acctgattgc cctgagcctg     1980

ggcctgaccc ccaacttcaa gagcaacttc gacctggccg aggatgccaa actgcagctg     2040

agcaaggaca cctacgacga cgacctggac aacctgctgg cccagatcgg cgaccagtac     2100

gccgacctgt ttctggccgc caagaacctg tccgacgcca tcctgctgag cgacatcctg     2160

agagtgaaca ccgagatcac caaggccccc ctgagcgcct ctatgatcaa gagatacgac     2220

gagcaccacc aggacctgac cctgctgaaa gctctcgtgc ggcagcagct gcctgagaag     2280

tacaaagaga ttttcttcga ccagagcaag aacggctacg ccggctacat tgacggcgga     2340

gccagccagg aagagttcta caagttcatc aagcccatcc tggaaaagat ggacggcacc     2400

gaggaactgc tcgtgaagct gaacagagag gacctgctgc ggaagcagcg gaccttcgac     2460

aacggcagca tcccccacca gatccacctg ggagagctgc acgccattct gcggcggcag     2520

gaagattttt acccattcct gaaggacaac cgggaaaaga tcgagaagat cctgaccttc     2580

cgcatcccct actacgtggg ccctctggcc aggggaaaca gcagattcgc ctggatgacc     2640

agaaagagcg aggaaaccat caccccctgg aacttcgagg aagtggtgga caagggcgct     2700

tccgcccaga gcttcatcga gcggatgacc aacttcgata agaacctgcc caacgagaag     2760

gtgctgccca agcacagcct gctgtacgag tacttcaccg tgtataacga gctgaccaaa     2820

gtgaaatacg tgaccgaggg aatgagaaag cccgccttcc tgagcggcga gcagaaaaag     2880

gccatcgtgg acctgctgtt caagaccaac cggaaagtga ccgtgaagca gctgaaagag     2940

gactacttca agaaaatcga gtgcttcgac tccgtggaaa tctccggcgt ggaagatcgg     3000

ttcaacgcct ccctgggcac ataccacgat ctgctgaaaa ttatcaagga caaggacttc     3060

ctggacaatg aggaaaacga ggacattctg gaagatatcg tgctgaccct gacactgttt     3120

gaggacagag agatgatcga ggaacggctg aaaacctatg cccacctgtt cgacgacaaa     3180

gtgatgaagc agctgaagcg gcggagatac accggctggg gcaggctgag ccggaagctg     3240

atcaacggca tccgggacaa gcagtccggc aagacaatcc tggatttcct gaagtccgac     3300

ggcttcgcca acagaaactt catgcagctg atccacgacg acagcctgac ctttaaagag     3360

gacatccaga aagcccaggt gtccggccag ggcgatagcc tgcacgagca cattgccaat     3420

ctggccggca gccccgccat taagaagggc atcctgcaga cagtgaaggt ggtggacgag     3480

ctcgtgaaag tgatgggccg gcacaagccc gagaacatcg tgatcgaaat ggccagagag     3540

aaccagacca cccagaaggg acagaagaac agccgcgaga gaatgaagcg gatcgaagag     3600

ggcatcaaag agctgggcag ccagatcctg aaagaacacc ccgtggaaaa cacccagctg     3660

cagaacgaga agctgtacct gtactacctg cagaatgggc gggatatgta cgtggaccag     3720

gaactggaca tcaaccggct gtccgactac gatgtggacc atatcgtgcc tcagagcttt     3780

ctgaaggacg actccatcga caacaaggtg ctgaccagaa gcgacaagaa ccggggcaag     3840

agcgacaacg tgccctccga agaggtcgtg aagaagatga agaactactg gcggcagctg     3900

ctgaacgcca agctgattac ccagagaaag ttcgacaatc tgaccaaggc cgagagaggc     3960

ggcctgagcg aactggataa ggccggcttc atcaagagac agctggtgga aacccggcag     4020

atcacaaagc acgtggcaca gatcctggac tcccggatga acactaagta cgacgagaat     4080

gacaagctga tccgggaagt gaaagtgatc accctgaagt ccaagctggt gtccgatttc     4140

cggaaggatt tccagtttta caaagtgcgc gagatcaaca actaccacca cgcccacgac     4200

gcctacctga acgccgtcgt gggaaccgcc ctgatcaaaa agtaccctaa gctggaaagc     4260

gagttcgtgt acggcgacta caaggtgtac gacgtgcgga agatgatcgc caagagcgag     4320

caggaaatcg gcaaggctac cgccaagtac ttcttctaca gcaacatcat gaactttttc     4380

aagaccgaga ttaccctggc caacggcgag atccggaagc ggcctctgat cgagacaaac     4440

ggcgaaaccg gggagatcgt gtgggataag ggccgggatt ttgccaccgt gcggaaagtg     4500

ctgagcatgc cccaagtgaa tatcgtgaaa aagaccgagg tgcagacagg cggcttcagc     4560

aaagagtcta tccggcccaa gaggaacagc gataagctga tcgccagaaa gaaggactgg     4620

gaccctaaga agtacggcgg cttcgtgagc cccaccgtgg cctattctgt gctggtggtg     4680

gccaaagtgg aaaagggcaa gtccaagaaa ctgaagagtg tgaaagagct gctggggatc     4740

accatcatgg aaagaagcag cttcgagaag aatcccatcg actttctgga agccaagggc     4800

tacaaagaag tgaaaaagga cctgatcatc aagctgccta agtactccct gttcgagctg     4860

gaaaacggcc ggaagagaat gctggcctct gcccggttcc tgcagaaggg aaacgaactg     4920

gccctgccct ccaaatatgt gaacttcctg tacctggcca gccactatga gaagctgaag     4980

ggctcccccg aggataatga gcagaaacag ctgtttgtgg aacagcacaa gcactacctg     5040

gacgagatca tcgagcagat cagcgagttc tccaagagag tgatcctggc cgacgctaat     5100

ctggacaaag tgctgtccgc ctacaacaag caccgggata agcccatcag agagcaggcc     5160

gagaatatca tccacctgtt taccctgacc aatctgggag cccctcgggc cttcaagtac     5220

tttgacacca ccatcgaccg gaaggtgtac cggagcacca aagaggtgct ggacgccacc     5280

ctgatccacc agagcatcac cggcctgtac gagacacgga tcgacctgtc tcagctggga     5340

ggtgactctg gcggctcaaa aagaaccgcc gacggcagcg aattcgagcc caagaagaag     5400

aggaaagtct aa                                                         5412


<210>  5
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hHBG.F primer

<400>  5
tgactgaatc ggaacaaggc aaagg                                             25


<210>  6
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hHBG.R primer

<400>  6
attcttcatc cctagccagc cgc                                               23


<210>  7
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  sgRNA-1

<400>  7
acccggaact ccctcaagca                                                   20


<210>  8
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  sgRNA-2

<400>  8
atttccatta gattcattag                                                   20


<210>  9
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  4.9kb Forward primer

<400>  9
acggataagt agatattgag gtaagc                                            26


<210>  10
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  4.9kb Reverse primer

<400>  10
gtctctttca gttagcagtg g                                                 21


<210>  11
<211>  27
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Taqman probe

<400>  11
actgcgctga aactgtggtc tttatga                                           27


<210>  12
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Proximal promoter

<400>  12
ctatctcaat gcaaatatct                                                   20


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

<220>
<223>  g22#1 (A)

<400>  13
agatatttgc attgagatag                                                   20


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

<220>
<223>  g22#2 (C)

<400>  14
gatatttgca ttgagatagt                                                   20


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

<220>
<223>  g22#3 (D)

<400>  15
cagatatttg cattgagata                                                   20


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

<220>
<223>  g22#4 (E)

<400>  16
acagatattt gcattgagat                                                   20


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

<220>
<223>  G22#5 (F)

<400>  17
gacagatatt tgcattgaga                                                   20


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

<220>
<223>  new KLF1 binding motif

<400>  18
cctcttgggg gccccttccc cac                                               23


<210>  19
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  complement to new KLF1 binding motif

<400>  19
ggagaacccc cggggaaggg gtg                                               23


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

<220>
<223>  new TAL1 binding motif

<400>  20
cactatctca atgcaaatat ct                                                22


<210>  21
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  complement to new TAL1 binding motif

<400>  21
gtgatagagt tacgtttata ga                                                22


<210>  22
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  new GATA1 binding motif

<400>  22
cttgaccaat agccttgaca agg                                               23


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

<220>
<223>  complement to new GATA1 binding motif

<400>  23
gaactggtta tcggaactgt tcc                                               23


<210>  24
<211>  45
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  GATA proximal to TAL1

<400>  24
cacactatct caatgcaaat atctgtctga aacggtccct ggcta                       45


<210>  25
<211>  45
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  complement to GATA proximal to TAL1

<400>  25
gtgtgataga gttacgttta tacacagact ttgccaggga ccgat                       45


<210>  26
<211>  35
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  GATA motif proximal to TAL1 binding site

<400>  26
ccccttcccc acactatctc aatgcaaaca tctgt                                  35


<210>  27
<211>  35
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  complement to GATA motif proximal to TAL1 binding site

<400>  27
ggggaagggg tgtgatagag ttacgtttgt agaca                                  35


<210>  28
<211>  35
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  HUDEP2 -175/-187&-189

<400>  28
ggggaagggg tgtggtggag ttacgtttgt agaca                                  35


