                         SEQUENCE LISTING

<110>  Children's Hospital of Philadelphia
 
<120>  COMPOSITIONS AND METHODS USEFUL FOR THE TREATMENT OF BONE MARROW 
       FAILURE DISEASES ASSOCIATED WITH RIBOSOMOPATHIES

<130>  CHOP-131-PCT

<150>  US 63/159,937
<151>  2021-03-11

<160>  50    

<170>  PatentIn version 3.5

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

<220>
<223>  Hectd1 FRTt F

<400>  1
tgcctgctat caagcctgat                                                   20


<210>  2
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Common_En2 R

<400>  2
ccaactgacc ttgggcaaga acat                                              24


<210>  3
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Hectd1 FRTt R

<400>  3
cagagagctg gcaggaaagt                                                   20


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

<220>
<223>  Hectd1 ES loxp F

<400>  4
tccagcttgg tatttgccca                                                   20


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

<220>
<223>  Hectd1 ES loxp R

<400>  5
actgatggcg agctcagacc                                                   20


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

<220>
<223>  Cre F

<400>  6
gcggtctggc agtaaaaact atc                                               23


<210>  7
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Cre R

<400>  7
gtgaaacagc attgctgtca ctt                                               23


<210>  8
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Internal control F

<400>  8
ctaggccaca gaattgaaag atct                                              24


<210>  9
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Internal Control R

<400>  9
gtaggtggaa attctagcat catcc                                             25


<210>  10
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Human HECTD1 F

<400>  10
ccagtgagag tgacacaggt                                                   20


<210>  11
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Human HECTD1 R

<400>  11
atcatcatcg agctcagcca                                                   20


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

<220>
<223>  Human ZNF622F

<400>  12
ggctcacatg accaaagacc                                                   20


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

<220>
<223>  Human ZNF622 R

<400>  13
cgttgcacca caagcaaatc                                                   20


<210>  14
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Human GAPDH F

<400>  14
ccacccatgg caaattcc                                                     18


<210>  15
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Human GAPDH R

<400>  15
tgggatttcc attgatgaca ag                                                22


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

<220>
<223>  Mouse Hectd1-exon3 pair1 F

<400>  16
agcccgtgcc ataacatact                                                   20


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

<220>
<223>  Mouse Hectd1-exon3 pair1 R

<400>  17
tctctgctgg tcctgttgtt                                                   20


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

<220>
<223>  Mouse Hectd1-exon3 pair2 F

<400>  18
actttcctgc cagctctctg                                                   20


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

<220>
<223>  Mouse Hectd1-exon3 pair2 R

<400>  19
ctatcctccg ggtgcattct                                                   20


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

<220>
<223>  Mouse Hectd1-exon6 F

<400>  20
ctggttgacc tcctcttggt                                                   20


<210>  21
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Hectd1-exon6 R

<400>  21
tctataagct gccgatggga                                                   20


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

<220>
<223>  Mouse Hectd1-exon22 F

<400>  22
gccgcttgga ggacatacta                                                   20


<210>  23
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Hectd1-exon22 R

<400>  23
agaccttcca tagccacgag                                                   20


<210>  24
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Hectd1-exon38 F

<400>  24
atgtacagcg gtcatgtgga                                                   20


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

<220>
<223>  Mouse Hectd1-exon38 R

<400>  25
gtcgcccatg cacataagtt                                                   20


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

<220>
<223>  Mouse ZNF622 F

<400>  26
agaggaggag ggagaggaag                                                   20


<210>  27
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse ZNF622 R

<400>  27
cactccaggg tcctcacatt                                                   20


<210>  28
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rps6 F

<400>  28
agctccgcac cttctatgag a                                                 21


<210>  29
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rps6 R

<400>  29
gggaaaacct tgcttgtcat tc                                                22


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

<220>
<223>  Mouse Rps7 F

<400>  30
agcgccaaga tcgtgaagc                                                    19


<210>  31
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rps7 R

<400>  31
caccaccaac ttcgatttcc tt                                                22


<210>  32
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rpl5 F

<400>  32
ggaggtgaat ggaggtgaat ac                                                22


<210>  33
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rpl5 R

<400>  33
agttgtagtt cgggcaagac                                                   20


<210>  34
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rpl11 F

<400>  34
atggcgcaag atcaagggg                                                    19


<210>  35
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Mouse Rpl11 R

<400>  35
gactgtgcag tgaacagcaa t                                                 21


<210>  36
<211>  1434
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Homo sapiens zinc finger protein 622

<400>  36
atggcgacgt acacctgcat aacttgccgg gtggcgttcc gcgacgcgga catgcagcgg       60

gcccactata agacggactg gcaccgctac aacctgcggc ggaaggtggc cagcatggcc      120

ccagtgaccg ccgagggctt ccaggagcga gtgcgggcgc agcgggccgt cgcggaggag      180

gagagcaagg gctcggccac ctactgcacc gtttgcagta agaagtttgc ctctttcaac      240

gcctacgaga accacctcaa gtcccggcgt cacgttgagc tggagaagaa ggccgtgcag      300

gcagtgaatc ggaaagtgga gatgatgaat gaaaagaact tggagaaagg actgggcgtg      360

gacagtgtgg acaaggatgc catgaacgcg gccatccagc aggccatcaa ggcccagccg      420

tccatgtctc ccaagaaggc gcccccagcg cctgcaaagg aggccaggaa tgtcgtggcc      480

gtgggtactg gtggccgtgg gacccacgac cgagacccga gtgagaaacc accccggctc      540

cagtggtttg aacagcaggc gaagaagttg gcaaagcagc aggaggagga cagcgaggag      600

gaggaagagg acctggatgg agacgattgg gaagatattg attctgatga agaattggaa      660

tgtgaggata ctgaagcaat ggacgatgtg gtggagcagg atgcagagga ggaagaggct      720

gaggaaggcc caccccttgg tgccatccct atcacggact gcttattttg ttcccatcat      780

tccagctcgc tgatgaagaa tgtggctcac atgaccaaag accacagttt ctttattcct      840

gatatagaat atctttcaga tattaaggga ctgattaaat acttgggaga gaaagttggt      900

gttggcaaga tttgcttgtg gtgcaacgag aaagggaagt ccttctactc cacagaagct      960

gtacaggcac atatgaatga caaaagccac tgtaagctct tcacagatgg cgatgctgct     1020

ttggaatttg cagacttcta tgattttagg agtagctatc cagatcacaa ggaaggggag     1080

gaccccaata aggctgagga gttgccctca gaaaagaact tggaatatga tgatgaaacc     1140

atggaattga ttctgccttc tggtgccaga gtgggtcatc gctccttgat gagatactac     1200

aaacagcgat ttggcttgtc aagagctgtg gcagttgcca aaaatcggaa ggccgtgggc     1260

cgagtacttc agcagtacag agccctggga tggactggca gcacaggagc ggctcttatg     1320

cgagagcgag acatgcagta tgtccaaagg atgaaatcaa aatggatgct gaagacagga     1380

atgaagaaca atgccaccaa gcagatgcac tttcgggtcc aagtgagatt ctga           1434


<210>  37
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  antisense1

<400>  37
tcatcatctc cactttccg                                                    19


<210>  38
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  shRNA1

<400>  38
caaagaccac agtttcttt                                                    19


<210>  39
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  shRNA2

<400>  39
accaaagacc acagtttctt ta                                                22


<210>  40
<211>  97
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  zinc finger protein 622 start position 466

<400>  40
tgctgttgac agtgagcgcc caaagaccac agtttcttta tagtgaagcc acagatgtat       60

aaagaaactg tggtctttgg ttgcctactg cctcgga                                97


<210>  41
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  antisense2

<400>  41
aaagaaactg tggtctttg                                                    19


<210>  42
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  shRNA3

<400>  42
caaagaccac agtttcttt                                                    19


<210>  43
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  shRNA4

<400>  43
accaaagacc acagtttctt ta                                                22


<210>  44
<211>  97
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  zinc finger protein 622 start position 952

<400>  44
tgctgttgac agtgagcgcc caaagaccac agtttcttta tagtgaagcc acagatgtat       60

aaagaaactg tggtctttgg ttgcctactg cctcgga                                97


<210>  45
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand1

<400>  45
cacctgcata acttgccggg tgg                                               23


<210>  46
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand2

<400>  46
ccgggtggcg ttccgcgacg cgg                                               23


<210>  47
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand3

<400>  47
gggcccacta taagacggac tgg                                               23


<210>  48
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand4

<400>  48
tactgaagca atggacgatg tgg                                               23


<210>  49
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand5

<400>  49
cgtgataggg atggcaccaa ggg                                               23


<210>  50
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  guide strand6

<400>  50
taagcagtcc gtgataggga tgg                                               23


