                         SEQUENCE LISTING

<110>  The Trustees of the University of Pennsylvania
       The Children's Hospital of Philadelphia
       Musunuru, Kiran
       Peranteau, William H.
       Morrisey, Edward
 
<120>  In Utero CRISPR-Mediated Therapeutic Editing of Genes

<130>  CHOP-105-PCT

<140>  PCT/US19/30083
<141>  2019-04-30

<150>  US 62/664,904
<151>  2018-04-30

<160>  125   

<170>  PatentIn version 3.5

<210>  1
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  1
ctggttccat gggaggctca agg                                               23


<210>  2
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  2
gaagtttcat tggatgctct ggg                                               23


<210>  3
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  3
caggttttat ggtatgctct agg                                               23


<210>  4
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  4
ccgcttcccg gggatgctct tgg                                               23


<210>  5
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  5
caggatggag gggatgctct tgg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  6
caagctctaa gggatgctct ggg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  7
tgttttccat gggatgctct tgg                                               23


<210>  8
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  8
cagtttccat ggggtgctct tgg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  9
cgcggtctat gggatgctct ggg                                               23


<210>  10
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  10
caggttccat gggatgctct ggg                                               23


<210>  11
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  11
ctttcatcag cacaaccacc tgg                                               23


<210>  12
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  12
catacaacat cacaaccatc agg                                               23


<210>  13
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  13
ccttcaccgt cacaaacacc tgg                                               23


<210>  14
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  14
cataaaatgt cacaaccaca tgg                                               23


<210>  15
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  15
aattcaaggt cacaaccccc agg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  16
atttcaaagt cacaaccaca ggg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  17
tattcaaaac cacaaccacc tgg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  18
ggagcaacgt cacaaccacc agg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  19
cggttaaggt cacaaccacc tgg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  20
cattcaacct cacaaccaca agg                                               23


<210>  21
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  21
cattcaacgt cacaaccacc agg                                               23


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

<220>
<223>  protospacer sequence

<400>  22
caggttccat gggatgctct                                                   20


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

<220>
<223>  target site sequence

<400>  23
aaacttttta aaaccaacct ttttcgaaat tataattaag ggttccggat                  50


<210>  24
<211>  49
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  24
aaattcgtat agcatacaca atacgaaaat atatttaagg gttccggat                   49


<210>  25
<211>  50
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  25
ataacttcgt atagcataca ttatacgaag ttatattaag ggttccggat                  50


<210>  26
<211>  47
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  26
aacttcgtat agcatacatt atacgaagtt atattaaggg ttccgat                     47


<210>  27
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  27
atcgtgcagg taagaaagca cctggg                                            26


<210>  28
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  28
aaaacctgca gttccaccgc ttctgg                                            26


<210>  29
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  29
tagctctctg cgatccattg tggtgg                                            26


<210>  30
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  30
gtgcaggtaa gaaagcacct ggg                                               23


<210>  31
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  31
acctgcagtt ccaccgcttc tgg                                               23


<210>  32
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  32
ctctctgcta tccattgtgg tgg                                               23


<210>  33
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  33
cctgcagatc ttcaccaaac cgg                                               23


<210>  34
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  34
catccagcgc cacaaccacc                                                   20


<210>  35
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  35
caccaggtac tgcttgtccc cgg                                               23


<210>  36
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer sequence

<400>  36
cattcaacgt cacaaccacc                                                   20


<210>  37
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  37
acattatacg aagttatatt aaggg                                             25


<210>  38
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  38
cattcaacgt cacaaccacc agg                                               23


<210>  39
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  39
gatccaggtg aaagagagca tgg                                               23


<210>  40
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  40
acctgcagtt ccaccggttc tgg                                               23


<210>  41
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  41
aaatgcagtc tgcctcagaa tgg                                               23


<210>  42
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  42
ggagcaagac aaatttggga agg                                               23


<210>  43
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  43
ttgtgcaggt gagaaacatt cgg                                               23


<210>  44
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  44
tgccaagcag gtagagaggc tgg                                               23


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

<220>
<223>  protospacer and PAM sequence

<400>  45
caacccagaa ggtcaccgag tgg                                               23


<210>  46
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  46
caggttccat gggatgctct                                                   20


<210>  47
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  47
cattcaacgt cacaaccacc                                                   20


<210>  48
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  48
ggtgctagcc ttgcgttccg                                                   20


<210>  49
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  protospacer and PAM sequence

<400>  49
acattatacg aagttatatt aa                                                22


<210>  50
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  50
tctcccccgc acttagtttc c                                                 21


<210>  51
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  51
ggactcagat gctgggctga tg                                                22


<210>  52
<211>  15
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mutated target sequence

<400>  52
gtcattcaac gtcac                                                        15


<210>  53
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  53
cctgtccgtt cgctttggaa g                                                 21


<210>  54
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  54
aaatctgtgc ggagccgaaa tc                                                22


<210>  55
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  55
agagagcaca gagaggtcag t                                                 21


<210>  56
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  56
ttcaggggct ggagttacgg                                                   20


<210>  57
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  57
catgtgtgga tgggggctta                                                   20


<210>  58
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  58
ctttggtggt gcagtagcct                                                   20


<210>  59
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  59
gcatacttga aggctgtgcc                                                   20


<210>  60
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  60
aatgtcactc cggcttctgt                                                   20


<210>  61
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  61
ccctgcggct aataaaccag a                                                 21


<210>  62
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  62
gaggatcctg tgtaacgggt g                                                 21


<210>  63
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  63
gccaccaaac ctgatgacct                                                   20


<210>  64
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  64
gctgttcagt taaccacggc                                                   20


<210>  65
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  65
cagacaccac ccccttttca                                                   20


<210>  66
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  66
ggtagctaga ggtgttgggc                                                   20


<210>  67
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  67
aagctttcag cgaggcatta                                                   20


<210>  68
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  68
ctgacatatg gatcagggcg t                                                 21


<210>  69
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  69
ccactgaaaa gcccttccct                                                   20


<210>  70
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  70
tgagtcccat tctcggaggt                                                   20


<210>  71
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  71
ctcagaagag agatgccgag g                                                 21


<210>  72
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  72
aaacagtgct aaacagtgac gc                                                22


<210>  73
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  73
tcgttgaaac ggagtgctga                                                   20


<210>  74
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  74
cattcataag gtacaatcaa cgaca                                             25


<210>  75
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  75
agttccggat gtctgaaggg a                                                 21


<210>  76
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  76
ttggccagct agaaagcaga                                                   20


<210>  77
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  77
gttagccaag gttgtacccc a                                                 21


<210>  78
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  78
catttggtgg tgtcagcctc                                                   20


<210>  79
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  79
cattccacac aacgcattcc                                                   20


<210>  80
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  80
acctcttgtg tctctaaggg c                                                 21


<210>  81
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  81
ccttccttta acagagccca ct                                                22


<210>  82
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  82
ggggtgagct gtccatgtg                                                    19


<210>  83
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  83
aaggaagcct ccccttgttt c                                                 21


<210>  84
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  84
aatgctgacc gccaagaaga                                                   20


<210>  85
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  85
ccatcccgtg accagatagc                                                   20


<210>  86
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  86
gctggctagc atggtgcat                                                    19


<210>  87
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  87
attgttcagc aacctgggga a                                                 21


<210>  88
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  88
aaggagaact ccctagcacc a                                                 21


<210>  89
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  89
catgaccagt gaccagtgtt g                                                 21


<210>  90
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  90
tcagtcatgt aacctccccc a                                                 21


<210>  91
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  91
aagactttgt gaaggctggg g                                                 21


<210>  92
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  92
aagcgcatac aacaagcaca                                                   20


<210>  93
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  93
gaatcagtgt cgcgtctagc                                                   20


<210>  94
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  94
cctcctgcag atcttcacca aaccgg                                            26


<210>  95
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  95
agtcatccag cgccacaacc accagg                                            26


<210>  96
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  96
aaccaccagg tactgcttgt ccccgg                                            26


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

<220>
<223>  target site sequence

<400>  97
accatcagcg ggccggtgag tatctggctg cactgagggc                             40


<210>  98
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  98
accatcagcg ggccggtgaa tatctggctg cactgagggc                             40


<210>  99
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  99
accatcagcg ggccggtaaa tatctggctg cactgagggc                             40


<210>  100
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  100
accatcagcg ggccggtaag tgtctggctg cactgagggc                             40


<210>  101
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  101
accatcagcg ggccggtgag tgtctggctg cactgagggc                             40


<210>  102
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  102
accatcagcg ggccggtagg tgtctggctg cactgagggc                             40


<210>  103
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  103
accatcagcg ggccggtggg tgtctggctg cactgagggc                             40


<210>  104
<211>  40
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target site sequence

<400>  104
accatcagcg ggccggtaag tatctggctg cactgagggc                             40


<210>  105
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  105
agctggcctt tcctagagtc                                                   20


<210>  106
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  106
cctggaagtc aaagtgggct                                                   20


<210>  107
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  107
ggctgccgga tgacaatgat                                                   20


<210>  108
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  108
gggtccatgc tacagcagtt                                                   20


<210>  109
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  109
cttatgcccc tgcattcctg at                                                22


<210>  110
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  110
cacatctcga cgaaaccgc                                                    19


<210>  111
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  111
gctcagggtc tttcgcctaa                                                   20


<210>  112
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  112
acacccagca caagagttga                                                   20


<210>  113
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  113
atgaaccaaa ccctcagcct                                                   20


<210>  114
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  114
agtctgattc aaataggaga atgtc                                             25


<210>  115
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  115
tgggtaagat ttcgcaggca                                                   20


<210>  116
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  116
ctgggcttga cacagattgg                                                   20


<210>  117
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  117
tctgaacctt cctgatgctc c                                                 21


<210>  118
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  118
ggcgtcctgg gatggttatt                                                   20


<210>  119
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  119
ccttcccacc acagggtttt                                                   20


<210>  120
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  120
tggccttgtg ctgtgaaact a                                                 21


<210>  121
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  121
ttcccatgga aattctagga gacc                                              24


<210>  122
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  122
ggaagtgggc agcactcaaa                                                   20


<210>  123
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  123
ggagaccttt ctgcccagtt g                                                 21


<210>  124
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  124
agaacagatt gcaagccatg aa                                                22


<210>  125
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  primer sequence

<400>  125
cttcctctgt ctggtgccat                                                   20


