                         SEQUENCE LISTING

<110>  The Children's Hospital of Philadelphia
 
<120>  IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF 
       MONOGENETIC DISEASES, INLCUDING MUCOPOLYSACCHARIDOSIS TYPE 1H AND
        OTHER DISORDERS

<130>  CHOP-127-PCT

<150>  US 63/056,954
<151>  2020-07-27

<160>  97    

<170>  PatentIn version 3.5

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

<220>
<223>  spCas9.ABEmax

<400>  1
gctctaggcc gaagtgtcgc agg                                               23


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

<220>
<223>  spCas9.ABEmax

<400>  2
taggccgaag tgtcgcaggc cgg                                               23


<210>  3
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nme2Cas9.ABEmax

<400>  3
gagcagctct aggccgaagt gtcgcaggcc                                        30


<210>  4
<211>  30
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nme2Cas9.ABEmax

<400>  4
ctctaggccg aagtgtcgca ggccgggacc                                        30


<210>  5
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SpG.ABEmax

<400>  5
ctctaggccg aagtgtcgca ggcc                                              24


<210>  6
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SpRY.ABEmax

<400>  6
ctctaggccg aagtgtcgca ggcc                                              24


<210>  7
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SauriCas9.ABEmax

<400>  7
agctctaggc cgaactctcg cagg                                              24


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

<220>
<223>  NRCH.ABEmax

<400>  8
gcagctctag gccgaagtgt cgca                                              24


<210>  9
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  E15.5 AAV.ABE.Idua-W392X

<400>  9
ctctaggcag aggtctcaaa gg                                                22


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

<220>
<223>  Exon:idua

<400>  10
actctaggca gaggtctcaa agg                                               23


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

<220>
<223>  Intron: 1700010l14Rik

<400>  11
gttctagact gaggtctcaa ggg                                               23


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

<220>
<223>  Intron:Wnt11

<400>  12
actccaagct ggggtctcaa cgg                                               23


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

<220>
<223>  Intergenic:Papl-Fbxo27

<400>  13
actctaggct agagtctcaa agg                                               23


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

<220>
<223>  Intergenic:Fgf9-Gm25614

<400>  14
acttttgaca gaggtatcaa ggg                                               23


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

<220>
<223>  Intergenic:Gm12106-Stc2

<400>  15
attccagcca gaggtatcaa agg                                               23


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

<220>
<223>  Intergenic:Gm26190-B3gat1

<400>  16
agttcagaca gaggtctcaa agg                                               23


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

<220>
<223>  Intergenic:Ccdc85c-Hhipl1

<400>  17
gctccaggca gaggtcccag ggg                                               23


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

<220>
<223>  Intron:Osbpl1a

<400>  18
gaactaagca gaggtctcaa agg                                               23


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

<220>
<223>  Intron:Blnk

<400>  19
gctctgagca gaggtcccaa cgg                                               23


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

<220>
<223>  IntronLRhoj

<400>  20
actctacaca gaggtaccaa tgg                                               23


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

<220>
<223>  ABE7.10 mRNA

<400>  21
ggcctagagc                                                              10


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

<220>
<223>  loxP targeting protospacer and PAM

<400>  22
attatacgaa gttatattaa ggg                                               23


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

<220>
<223>  loxP targeting protospacer

<400>  23
attatacgaa gttatattaa                                                   20


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

<220>
<223>  Mouse IDUA protospacer

<400>  24
actctaggca gaggtctcaa                                                   20


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

<220>
<223>  Primer Idua-F

<400>  25
tgctaggtat gagagagcca                                                   20


<210>  26
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Idua-R

<400>  26
agtgtagatg aggactgtgg t                                                 21


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

<220>
<223>  Primer Intron:1700010I14Rik F

<400>  27
gggattgctc tgctctgtct                                                   20


<210>  28
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:1700010I14Rik R

<400>  28
tgtgtaagag tgggccatgt                                                   20


<210>  29
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Wnt11 F

<400>  29
caggcttgaa cacacacaca                                                   20


<210>  30
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Wnt11 R

<400>  30
aaaatcccgt tgagacccca                                                   20


<210>  31
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Papl-Fbxo27 F

<400>  31
caacatttgg aagtctgagg c                                                 21


<210>  32
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Papl-Fbxo27 R

<400>  32
tgctggggtt acaagggtg                                                    19


<210>  33
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Fgf9-Gm25614 F

<400>  33
actgcaggaa tggaaaactc c                                                 21


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

<220>
<223>  Primer Intergenic:Fgf9-Gm25614 R

<400>  34
ctctagagac cctgtgctgg                                                   20


<210>  35
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Gm12106-Stc2 F

<400>  35
aggccttcga tcagacatca                                                   20


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

<220>
<223>  Primer Intergenic:Gm12106-Stc2 R

<400>  36
caacaacatg gctgctcagg                                                   20


<210>  37
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Gm26190-B3gat1 F

<400>  37
ccttcactct cttgggcctt                                                   20


<210>  38
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Gm26190-B3gat1 R

<400>  38
cagtgtcagc aaagggaagc                                                   20


<210>  39
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Ccdc85c-Hhipl1 F

<400>  39
acaaggaggg gtgtgtgtac                                                   20


<210>  40
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intergenic:Ccdc85c-Hhipl1 R

<400>  40
ctgctgagag gtcctggag                                                    19


<210>  41
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Osbpl1a F

<400>  41
gcccacttaa taaccctgtg t                                                 21


<210>  42
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Osbpl1a R

<400>  42
gcaggagggg tcattgatct                                                   20


<210>  43
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Blnk F

<400>  43
acagcactga gaagggacaa                                                   20


<210>  44
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Blnk R

<400>  44
cgggagggat cgtaaagtga                                                   20


<210>  45
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer Intron:Rhoj F

<400>  45
ttggctagtc tccgtgtgaa                                                   20


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

<220>
<223>  Primer Intron:Rhoj R

<400>  46
ggggtctaga ggtctttggg                                                   20


<210>  47
<211>  17
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Primer CAIDUA_F

<400>  47
ctggcgctgc tggccgc                                                      17


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

<220>
<223>  Primer CAIDUA_R

<400>  48
ctggacggtc ccgggccctg                                                   20


<210>  49
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  dMPS1.1

<400>  49
ttctgatgag ggctccgcgg                                                   20


<210>  50
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  dMPS1.2

<400>  50
gcttctgatg agggctccgc                                                   20


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

<220>
<223>  dMPS1.3

<400>  51
ggcttctgat gagggctccg                                                   20


<210>  52
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  dMPS1.4

<400>  52
cttctgatga gggctccgcg                                                   20


<210>  53
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  dMPS4

<400>  53
tctgatgagg gctccgcggg                                                   20


<210>  54
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  human IDUA protospacer

<400>  54
gctctaggcc gaagtgtcgc                                                   20


<210>  55
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Hu.IDU-F

<400>  55
caatgccttc ctgagctacc ac                                                22


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

<220>
<223>  Hu.IDUA-R

<400>  56
aggtagcgcg tgacgtagac                                                   20


<210>  57
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr10 Exon: MIR3663HG

<400>  57
gctctgggct ggggtgtcgc tgg                                               23


<210>  58
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr8 Intergenic: MIR3686-RP11-419K12.1

<400>  58
gctctaggct gaagtgcttc tgg                                               23


<210>  59
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr19 Intergenic: RAB11B-AS1/RAB11B-RAB11B

<400>  59
ccactaggcc aaagtgtagc tgg                                               23


<210>  60
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr3 Intergenic: RBM15B/VPRBP-VPRBP

<400>  60
gctccaggag gaagtgtcac agg                                               23


<210>  61
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr8 Intron: DLC1

<400>  61
gttctaggtg gaagtgttgc tgg                                               23


<210>  62
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr17 Intergenic: ASIC2-AA06

<400>  62
ggtctaggcc aagctgtcgc tgg                                               23


<210>  63
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ChrX Intron: LINC01456

<400>  63
gctctaggca gaagagttga cgg                                               23


<210>  64
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr22 Intergenic: IGLV10-54-VPREB1

<400>  64
gatcaaggct gaagtgtccc tgg                                               23


<210>  65
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr4 Intergenic: RP11-1398P2.1-FAM53A

<400>  65
gctccaggcc caaatgtccc tgg                                               23


<210>  66
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr13 Intergenic: ENSG279924

<400>  66
gctctaggcc gaagtgtcgc agg                                               23


<210>  67
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr1 Intergenic: CCDC17-GPBP1L1

<400>  67
gctctaggcc gaagaggcgg gag                                               23


<210>  68
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr14 Intron: FUT8

<400>  68
actataggct gaagtgtcct ggg                                               23


<210>  69
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr22 Exon: GRAMD4

<400>  69
gctctagaac gaagttcaca ggt                                               23


<210>  70
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr3 Intron: KALRN

<400>  70
tcactaggcc aaagtgtatc tgg                                               23


<210>  71
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr18 Intron: L3MBTL4

<400>  71
tctctaggtc agaagtgtca ctg                                               23


<210>  72
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr12 Intergenic: LINC02368-LOC107987176

<400>  72
gctttaggca gaagtgtcaa gga                                               23


<210>  73
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr5 Intergenic: PPIGP1-SGCD

<400>  73
gctctaaggc caaagtgtgc ttg                                               23


<210>  74
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr14 Intergenic: BEGAIN-LINC00523

<400>  74
tcactaggca aagtgtcaca ggc                                               23


<210>  75
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr7 Intron: KLHDC10

<400>  75
gctccaggca aagtgtctaa ggg                                               23


<210>  76
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr4 Intron: MAN2B2

<400>  76
gctctatgcc gaagtgttcg gaa                                               23


<210>  77
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr13 Intron: FOXO1

<400>  77
gctcaggccg gagtgtcgtg gca                                               23


<210>  78
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr19 Intergenic: LINC01801

<400>  78
agactaggct gaagtgtccc agg                                               23


<210>  79
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr19 Intergenic: DAND5-NFIX

<400>  79
catctagggg aagtgtcact ggg                                               23


<210>  80
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr15 Intron: ARNT2

<400>  80
actctaggtc aaagtgtcag ctg                                               23


<210>  81
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr16 Intron: GSE1

<400>  81
actctaggcc caatgtccca ggg                                               23


<210>  82
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr1 Unchar: LOC105378839

<400>  82
tctctaggac aaagttgtct cag                                               23


<210>  83
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr5 Intergenic: LOC105377702-LINC01938

<400>  83
gctctattag aagtgtagca gga                                               23


<210>  84
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr3 Intron: SOX2-OT

<400>  84
tctctaggcc gaagtcttag gga                                               23


<210>  85
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr20 Unchar: LOC105372578

<400>  85
tctctaggct gaagtgccct ggg                                               23


<210>  86
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr13 Intergenic: MIR5007-HNF4GP1

<400>  86
tctctaagga agtgtcacag gag                                               23


<210>  87
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr1 Intron: WDR47

<400>  87
gctctagacc aaagtggtct agg                                               23


<210>  88
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr10 Intron: PARD3

<400>  88
gctctagggc caaaggtcac agg                                               23


<210>  89
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr20 Intergenic: SLC2A10-RN7SKP33

<400>  89
gctctaagca gtagtgtgct ggt                                               23


<210>  90
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr4 Intergenic: LOC105377476-MIR584G

<400>  90
tctctaggca gaagtgatgc tgg                                               23


<210>  91
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr18 Intron: ZBTB7C

<400>  91
gctctaggca ggagggtccc tcc                                               23


<210>  92
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr1 Intron: TRABD2B

<400>  92
gctctaagga agtgtggcag gtg                                               23


<210>  93
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr10 Exon: CHT25H

<400>  93
actccatgtc gaagagtagc agg                                               23


<210>  94
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr17 Intron: Septin9

<400>  94
gctctaagat ggagtgtccc agg                                               23


<210>  95
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr2 Intergenic: HSPE1P9-ARL4C

<400>  95
actctaggaa gaagtgactc agg                                               23


<210>  96
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Chr3 Intron: NEC11

<400>  96
tctctaggcc taagtgtgga aag                                               23


<210>  97
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ABE7.10mRNA

<400>  97
gcgacacttc ggcctagagc tcc                                               23


