JP2018520149A5 - - Google Patents
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- JP2018520149A5 JP2018520149A5 JP2017567629A JP2017567629A JP2018520149A5 JP 2018520149 A5 JP2018520149 A5 JP 2018520149A5 JP 2017567629 A JP2017567629 A JP 2017567629A JP 2017567629 A JP2017567629 A JP 2017567629A JP 2018520149 A5 JP2018520149 A5 JP 2018520149A5
- Authority
- JP
- Japan
- Prior art keywords
- combination
- composition
- corneal dystrophy
- nuclease
- dystrophy
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 239000000203 mixture Substances 0.000 claims description 48
- 101710163270 Nuclease Proteins 0.000 claims description 43
- 206010011005 corneal dystrophy Diseases 0.000 claims description 38
- 206010008025 Cerebellar ataxia Diseases 0.000 claims description 34
- 108020004414 DNA Proteins 0.000 claims description 24
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims description 20
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 20
- 230000008439 repair process Effects 0.000 claims description 16
- 230000008685 targeting Effects 0.000 claims description 14
- 208000036626 Mental retardation Diseases 0.000 claims description 12
- 208000035475 disorder Diseases 0.000 claims description 12
- 230000005782 double-strand break Effects 0.000 claims description 12
- 201000003775 lattice corneal dystrophy Diseases 0.000 claims description 12
- 210000001519 tissue Anatomy 0.000 claims description 12
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 8
- 102000007371 Ataxin-3 Human genes 0.000 claims description 8
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 claims description 8
- 108020005004 Guide RNA Proteins 0.000 claims description 8
- 208000023105 Huntington disease Diseases 0.000 claims description 8
- 208000002569 Machado-Joseph Disease Diseases 0.000 claims description 8
- 108091092878 Microsatellite Proteins 0.000 claims description 8
- 208000036834 Spinocerebellar ataxia type 3 Diseases 0.000 claims description 8
- 201000010099 disease Diseases 0.000 claims description 8
- 239000013604 expression vector Substances 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 208000011580 syndromic disease Diseases 0.000 claims description 8
- 230000003205 diastolic effect Effects 0.000 claims description 7
- 206010003591 Ataxia Diseases 0.000 claims description 6
- 208000033051 Fuchs endothelial corneal dystrophy Diseases 0.000 claims description 6
- 230000006780 non-homologous end joining Effects 0.000 claims description 6
- 210000004087 cornea Anatomy 0.000 claims description 5
- 230000035772 mutation Effects 0.000 claims description 5
- 102100024378 AF4/FMR2 family member 2 Human genes 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 206010066131 Congenital central hypoventilation syndrome Diseases 0.000 claims description 4
- 102000053602 DNA Human genes 0.000 claims description 4
- 206010058314 Dysplasia Diseases 0.000 claims description 4
- 206010015995 Eyelid ptosis Diseases 0.000 claims description 4
- 208000001914 Fragile X syndrome Diseases 0.000 claims description 4
- 206010056438 Growth hormone deficiency Diseases 0.000 claims description 4
- 101000833172 Homo sapiens AF4/FMR2 family member 2 Proteins 0.000 claims description 4
- 101000828537 Homo sapiens Synaptic functional regulator FMR1 Proteins 0.000 claims description 4
- 206010021750 Infantile Spasms Diseases 0.000 claims description 4
- 208000035899 Infantile spasms syndrome Diseases 0.000 claims description 4
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 4
- 201000003692 Partington syndrome Diseases 0.000 claims description 4
- 102100023532 Synaptic functional regulator FMR1 Human genes 0.000 claims description 4
- 206010044565 Tremor Diseases 0.000 claims description 4
- 201000006791 West syndrome Diseases 0.000 claims description 4
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 201000004889 corneal granular dystrophy Diseases 0.000 claims description 4
- 206010015037 epilepsy Diseases 0.000 claims description 4
- 208000014706 granular corneal dystrophy Diseases 0.000 claims description 4
- 238000002347 injection Methods 0.000 claims description 4
- 239000007924 injection Substances 0.000 claims description 4
- 201000006938 muscular dystrophy Diseases 0.000 claims description 4
- 208000003580 polydactyly Diseases 0.000 claims description 4
- 210000004129 prosencephalon Anatomy 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 102000004169 proteins and genes Human genes 0.000 claims description 4
- 201000003004 ptosis Diseases 0.000 claims description 4
- 230000001850 reproductive effect Effects 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- 210000003625 skull Anatomy 0.000 claims description 4
- 206010010356 Congenital anomaly Diseases 0.000 claims description 3
- 206010064571 Gene mutation Diseases 0.000 claims description 3
- 201000004173 Epithelial basement membrane dystrophy Diseases 0.000 claims description 2
- 201000004216 Posterior amorphous corneal dystrophy Diseases 0.000 claims description 2
- 241000700605 Viruses Species 0.000 claims description 2
- 206010002022 amyloidosis Diseases 0.000 claims description 2
- 230000003511 endothelial effect Effects 0.000 claims description 2
- 208000001901 epithelial recurrent erosion dystrophy Diseases 0.000 claims description 2
- 210000000744 eyelid Anatomy 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims description 2
- 238000001361 intraarterial administration Methods 0.000 claims description 2
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 238000007912 intraperitoneal administration Methods 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 210000003786 sclera Anatomy 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- 208000009996 subepithelial mucinous corneal dystrophy Diseases 0.000 claims description 2
- 238000002054 transplantation Methods 0.000 claims description 2
- 210000001508 eye Anatomy 0.000 claims 14
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims 2
- 210000001638 cerebellum Anatomy 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 201000001922 Chandler syndrome Diseases 0.000 claims 1
- 201000004182 Congenital stromal corneal dystrophy Diseases 0.000 claims 1
- 206010010996 Corneal degeneration Diseases 0.000 claims 1
- 210000005252 bulbus oculi Anatomy 0.000 claims 1
- 210000002555 descemet membrane Anatomy 0.000 claims 1
- 201000005139 macular corneal dystrophy Diseases 0.000 claims 1
- 210000004379 membrane Anatomy 0.000 claims 1
- 239000012528 membrane Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 32
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 230000009467 reduction Effects 0.000 description 2
- 208000002320 spinal muscular atrophy Diseases 0.000 description 2
- 201000004175 X-linked endothelial corneal dystrophy Diseases 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000010412 perfusion Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562188013P | 2015-07-02 | 2015-07-02 | |
| US62/188,013 | 2015-07-02 | ||
| PCT/US2016/040962 WO2017004616A1 (en) | 2015-07-02 | 2016-07-05 | Crispr/cas9-based treatments |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018520149A JP2018520149A (ja) | 2018-07-26 |
| JP2018520149A5 true JP2018520149A5 (enExample) | 2019-08-15 |
Family
ID=57609222
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017567629A Pending JP2018520149A (ja) | 2015-07-02 | 2016-07-05 | Ｃｒｉｓｐｒ／ｃａｓ９をベースとした治療 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200010854A1 (enExample) |
| EP (1) | EP3317409A4 (enExample) |
| JP (1) | JP2018520149A (enExample) |
| KR (1) | KR20180041120A (enExample) |
| CN (1) | CN108350446A (enExample) |
| AU (1) | AU2016287836A1 (enExample) |
| BR (1) | BR112017028201A2 (enExample) |
| CA (1) | CA2989331A1 (enExample) |
| CL (1) | CL2017003411A1 (enExample) |
| EA (1) | EA201890203A1 (enExample) |
| IL (1) | IL256279A (enExample) |
| MX (1) | MX2017016921A (enExample) |
| WO (1) | WO2017004616A1 (enExample) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| CN106852157B (zh) | 2014-06-16 | 2022-04-12 | 约翰斯·霍普金斯大学 | 用于使用h1启动子表达crispr向导rna的组合物和方法 |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| EP3374502B1 (en) * | 2015-11-13 | 2021-10-27 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| CN109414450A (zh) * | 2016-04-22 | 2019-03-01 | 因特利亚治疗公司 | 用于治疗与转录因子4中三核苷酸重复相关的疾病的组合物和方法 |
| AU2017306676B2 (en) | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| US11512312B2 (en) | 2017-03-10 | 2022-11-29 | The Board Of Regents Of The University Of Texas System | Treatment of Fuchs' endothelial corneal dystrophy |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| JP7161730B2 (ja) * | 2017-06-07 | 2022-10-27 | 国立大学法人 東京大学 | 顆粒状角膜変性症に対する遺伝子治療薬 |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| DE102017131324A1 (de) | 2017-12-27 | 2019-06-27 | Beckhoff Automation Gmbh | Statormodul und Planarantriebssystem |
| CN108949823B (zh) * | 2017-12-29 | 2020-02-14 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 靶向敲除ATXN3基因中扩展突变型polyQ序列的方法 |
| WO2019217294A1 (en) | 2018-05-06 | 2019-11-14 | Emendobio Inc. | Differential knockout of an allele of a heterozygous elane gene |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| WO2020014261A1 (en) | 2018-07-09 | 2020-01-16 | The Broad Institute, Inc. | Rna programmable epigenetic rna modifiers and uses thereof |
| KR102126573B1 (ko) | 2018-10-18 | 2020-06-26 | 대한민국 | CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도 |
| WO2020088450A1 (zh) * | 2018-10-29 | 2020-05-07 | 中国农业大学 | 新型CRISPR/Cas12f酶和系统 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| BR112021018607A2 (pt) | 2019-03-19 | 2021-11-23 | Massachusetts Inst Technology | Métodos e composições para editar sequências de nucleotídeos |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021076883A2 (en) * | 2019-10-16 | 2021-04-22 | Brown University | Muscle regeneration and growth |
| AU2021267940A1 (en) | 2020-05-08 | 2022-12-08 | President And Fellows Of Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| CN111849991B (zh) * | 2020-08-05 | 2022-04-08 | 武汉纽福斯生物科技有限公司 | 一种寡核苷酸及其应用 |
| US20250319202A1 (en) * | 2021-11-09 | 2025-10-16 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| WO2023092132A1 (en) * | 2021-11-22 | 2023-05-25 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| IL320664A (en) * | 2022-11-02 | 2025-07-01 | Emendobio Inc | Compositions and methods for transcription factor 4 (tcf4) repeat expansion excision |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014093655A2 (en) * | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| MX374529B (es) * | 2013-12-12 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma. |
| EP3080257A1 (en) * | 2013-12-12 | 2016-10-19 | The Broad Institute Inc. | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
| CN109414450A (zh) * | 2016-04-22 | 2019-03-01 | 因特利亚治疗公司 | 用于治疗与转录因子4中三核苷酸重复相关的疾病的组合物和方法 |
-
2016
- 2016-07-05 KR KR1020187002971A patent/KR20180041120A/ko not_active Withdrawn
- 2016-07-05 JP JP2017567629A patent/JP2018520149A/ja active Pending
- 2016-07-05 BR BR112017028201A patent/BR112017028201A2/pt not_active Application Discontinuation
- 2016-07-05 EP EP16818953.8A patent/EP3317409A4/en not_active Withdrawn
- 2016-07-05 CA CA2989331A patent/CA2989331A1/en not_active Abandoned
- 2016-07-05 US US15/741,444 patent/US20200010854A1/en not_active Abandoned
- 2016-07-05 CN CN201680050474.4A patent/CN108350446A/zh active Pending
- 2016-07-05 AU AU2016287836A patent/AU2016287836A1/en not_active Abandoned
- 2016-07-05 MX MX2017016921A patent/MX2017016921A/es unknown
- 2016-07-05 WO PCT/US2016/040962 patent/WO2017004616A1/en not_active Ceased
- 2016-07-05 EA EA201890203A patent/EA201890203A1/ru unknown
-
2017
- 2017-12-12 IL IL256279A patent/IL256279A/en unknown
- 2017-12-27 CL CL2017003411A patent/CL2017003411A1/es unknown
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