JP2019507579A5 - - Google Patents
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- JP2019507579A5 JP2019507579A5 JP2018521542A JP2018521542A JP2019507579A5 JP 2019507579 A5 JP2019507579 A5 JP 2019507579A5 JP 2018521542 A JP2018521542 A JP 2018521542A JP 2018521542 A JP2018521542 A JP 2018521542A JP 2019507579 A5 JP2019507579 A5 JP 2019507579A5
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- 238000000034 method Methods 0.000 claims description 117
- 239000000203 mixture Substances 0.000 claims description 108
- 108010069091 Dystrophin Proteins 0.000 claims description 86
- 108700024394 Exon Proteins 0.000 claims description 57
- 230000005783 single-strand break Effects 0.000 claims description 44
- 230000005782 double-strand break Effects 0.000 claims description 39
- 238000003780 insertion Methods 0.000 claims description 33
- 230000037431 insertion Effects 0.000 claims description 33
- 210000004027 cell Anatomy 0.000 claims description 32
- 239000002299 complementary DNA Substances 0.000 claims description 28
- 102000001039 Dystrophin Human genes 0.000 claims description 27
- 238000012217 deletion Methods 0.000 claims description 26
- 230000037430 deletion Effects 0.000 claims description 26
- 239000000370 acceptor Substances 0.000 claims description 24
- 102000040430 polynucleotide Human genes 0.000 claims description 23
- 108091033319 polynucleotide Proteins 0.000 claims description 23
- 239000002157 polynucleotide Substances 0.000 claims description 23
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 20
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 20
- 108020004414 DNA Proteins 0.000 claims description 17
- 102000053602 DNA Human genes 0.000 claims description 17
- 230000008439 repair process Effects 0.000 claims description 17
- 125000006850 spacer group Chemical group 0.000 claims description 16
- 102000004533 Endonucleases Human genes 0.000 claims description 15
- 108010042407 Endonucleases Proteins 0.000 claims description 15
- 108091033409 CRISPR Proteins 0.000 claims description 14
- 230000000295 complement effect Effects 0.000 claims description 14
- 229920002477 rna polymer Polymers 0.000 claims description 13
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 12
- 239000012634 fragment Substances 0.000 claims description 12
- 230000000694 effects Effects 0.000 claims description 11
- 108090000623 proteins and genes Proteins 0.000 claims description 11
- 108091092195 Intron Proteins 0.000 claims description 10
- 230000001594 aberrant effect Effects 0.000 claims description 10
- 239000000463 material Substances 0.000 claims description 9
- 210000003205 muscle Anatomy 0.000 claims description 9
- 108020005004 Guide RNA Proteins 0.000 claims description 8
- 101100351033 Mus musculus Pax7 gene Proteins 0.000 claims description 8
- 239000013611 chromosomal DNA Substances 0.000 claims description 8
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 8
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims description 8
- 150000002632 lipids Chemical class 0.000 claims description 8
- 239000002105 nanoparticle Substances 0.000 claims description 8
- 210000001082 somatic cell Anatomy 0.000 claims description 8
- 210000000130 stem cell Anatomy 0.000 claims description 7
- 238000003776 cleavage reaction Methods 0.000 claims description 6
- 210000005260 human cell Anatomy 0.000 claims description 6
- 108020004999 messenger RNA Proteins 0.000 claims description 6
- 230000007017 scission Effects 0.000 claims description 6
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- -1 NANOG Proteins 0.000 claims description 4
- 210000000663 muscle cell Anatomy 0.000 claims description 4
- 239000013598 vector Substances 0.000 claims description 4
- 210000003130 muscle precursor cell Anatomy 0.000 claims description 3
- 102000004169 proteins and genes Human genes 0.000 claims description 3
- 101150018129 CSF2 gene Proteins 0.000 claims description 2
- 101150069031 CSN2 gene Proteins 0.000 claims description 2
- 108020004705 Codon Proteins 0.000 claims description 2
- 101150074775 Csf1 gene Proteins 0.000 claims description 2
- 101100239628 Danio rerio myca gene Proteins 0.000 claims description 2
- 101150106478 GPS1 gene Proteins 0.000 claims description 2
- 101001139134 Homo sapiens Krueppel-like factor 4 Proteins 0.000 claims description 2
- 101000687905 Homo sapiens Transcription factor SOX-2 Proteins 0.000 claims description 2
- 102100020677 Krueppel-like factor 4 Human genes 0.000 claims description 2
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 claims description 2
- 101100385413 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) csm-3 gene Proteins 0.000 claims description 2
- 102100035423 POU domain, class 5, transcription factor 1 Human genes 0.000 claims description 2
- 101710126211 POU domain, class 5, transcription factor 1 Proteins 0.000 claims description 2
- 101100047461 Rattus norvegicus Trpm8 gene Proteins 0.000 claims description 2
- 102100024270 Transcription factor SOX-2 Human genes 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 230000002159 abnormal effect Effects 0.000 claims description 2
- 101150055766 cat gene Proteins 0.000 claims description 2
- 101150055601 cops2 gene Proteins 0.000 claims description 2
- 238000004520 electroporation Methods 0.000 claims description 2
- 210000002950 fibroblast Anatomy 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims description 2
- 238000010362 genome editing Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 101150111214 lin-28 gene Proteins 0.000 claims description 2
- 230000001114 myogenic effect Effects 0.000 claims description 2
- 230000002018 overexpression Effects 0.000 claims description 2
- 210000001778 pluripotent stem cell Anatomy 0.000 claims description 2
- 229920001184 polypeptide Polymers 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 210000002966 serum Anatomy 0.000 claims description 2
- 229940126586 small molecule drug Drugs 0.000 claims description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 3
- 101710163270 Nuclease Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 108700029631 X-Linked Genes Proteins 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 210000001766 X chromosome Anatomy 0.000 description 1
- 230000004952 protein activity Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021097644A JP7684106B2 (ja) | 2015-10-28 | 2021-03-25 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
| JP2022212927A JP2023037643A (ja) | 2015-10-28 | 2022-12-14 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562247484P | 2015-10-28 | 2015-10-28 | |
| US62/247,484 | 2015-10-28 | ||
| US201662324064P | 2016-04-18 | 2016-04-18 | |
| US62/324,064 | 2016-04-18 | ||
| PCT/IB2016/001679 WO2017072590A1 (en) | 2015-10-28 | 2016-10-28 | Materials and methods for treatment of duchenne muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021097644A Division JP7684106B2 (ja) | 2015-10-28 | 2021-03-25 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019507579A JP2019507579A (ja) | 2019-03-22 |
| JP2019507579A5 true JP2019507579A5 (cg-RX-API-DMAC7.html) | 2020-02-20 |
Family
ID=57708608
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521542A Withdrawn JP2019507579A (ja) | 2015-10-28 | 2016-10-28 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
| JP2021097644A Active JP7684106B2 (ja) | 2015-10-28 | 2021-03-25 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
| JP2022212927A Withdrawn JP2023037643A (ja) | 2015-10-28 | 2022-12-14 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021097644A Active JP7684106B2 (ja) | 2015-10-28 | 2021-03-25 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
| JP2022212927A Withdrawn JP2023037643A (ja) | 2015-10-28 | 2022-12-14 | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (3) | US11369692B2 (cg-RX-API-DMAC7.html) |
| EP (3) | EP3368063B1 (cg-RX-API-DMAC7.html) |
| JP (3) | JP2019507579A (cg-RX-API-DMAC7.html) |
| CN (1) | CN108513546A (cg-RX-API-DMAC7.html) |
| AU (3) | AU2016344609B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3000931A1 (cg-RX-API-DMAC7.html) |
| DK (1) | DK4279084T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES3040945T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017072590A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (88)
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| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
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| US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
| SG10202104041PA (en) | 2015-10-23 | 2021-06-29 | Harvard College | Nucleobase editors and uses thereof |
| BR112018008971A2 (pt) | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| EP3384055B1 (en) | 2015-11-30 | 2025-07-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
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2016
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- 2016-10-28 EP EP16820319.8A patent/EP3368063B1/en active Active
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- 2016-10-28 WO PCT/IB2016/001679 patent/WO2017072590A1/en not_active Ceased
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- 2016-10-28 JP JP2018521542A patent/JP2019507579A/ja not_active Withdrawn
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