JP2018516978A5 - - Google Patents
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- JP2018516978A5 JP2018516978A5 JP2018511592A JP2018511592A JP2018516978A5 JP 2018516978 A5 JP2018516978 A5 JP 2018516978A5 JP 2018511592 A JP2018511592 A JP 2018511592A JP 2018511592 A JP2018511592 A JP 2018511592A JP 2018516978 A5 JP2018516978 A5 JP 2018516978A5
- Authority
- JP
- Japan
- Prior art keywords
- guide rna
- cas
- cas protein
- mutation
- promoter
- 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.)
- Granted
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Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021189812A JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A JP7814430B2 (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562162720P | 2015-05-16 | 2015-05-16 | |
| US62/162,720 | 2015-05-16 | ||
| PCT/US2016/027987 WO2016186772A2 (en) | 2015-05-16 | 2016-04-15 | Gene editing of deep intronic mutations |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021189812A Division JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018516978A JP2018516978A (ja) | 2018-06-28 |
| JP2018516978A5 true JP2018516978A5 (enExample) | 2019-05-23 |
| JP6985250B2 JP6985250B2 (ja) | 2021-12-22 |
Family
ID=55809259
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018511592A Active JP6985250B2 (ja) | 2015-05-16 | 2016-04-15 | 深部イントロン突然変異の遺伝子編集 |
| JP2021189812A Pending JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A Active JP7814430B2 (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021189812A Pending JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A Active JP7814430B2 (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11896651B2 (enExample) |
| EP (1) | EP3298134B8 (enExample) |
| JP (3) | JP6985250B2 (enExample) |
| KR (2) | KR20230142649A (enExample) |
| CN (3) | CN114657180A (enExample) |
| AU (2) | AU2016265255B2 (enExample) |
| CA (1) | CA2986021A1 (enExample) |
| ES (1) | ES2952598T3 (enExample) |
| IL (2) | IL296365A (enExample) |
| MX (2) | MX2017014665A (enExample) |
| PL (1) | PL3298134T3 (enExample) |
| PT (1) | PT3298134T (enExample) |
| RU (2) | RU2021132397A (enExample) |
| SG (2) | SG10201910750WA (enExample) |
| WO (1) | WO2016186772A2 (enExample) |
Families Citing this family (77)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| CA2948728A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| CA3276269A1 (en) | 2014-03-18 | 2026-03-02 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015148863A2 (en) | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3126495A1 (en) | 2014-04-02 | 2017-02-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CA2968440A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| EP3286571B1 (en) | 2015-04-24 | 2021-08-18 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| RU2021132397A (ru) * | 2015-05-16 | 2022-02-24 | Джензим Корпорейшн | Генное редактирование глубоких интронных мутаций |
| EP3310369B1 (en) * | 2015-06-19 | 2022-05-04 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| SG10202104041PA (en) | 2015-10-23 | 2021-06-29 | Harvard College | Nucleobase editors and uses thereof |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| US20170332610A1 (en) | 2016-05-20 | 2017-11-23 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| CA3032822A1 (en) * | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| IT201600102542A1 (it) * | 2016-10-12 | 2018-04-12 | Univ Degli Studi Di Trento | Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza. |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| WO2018106693A1 (en) | 2016-12-05 | 2018-06-14 | Editas Medicine, Inc. | SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | 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 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2018176009A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| EP3622073A4 (en) * | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| 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 |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| US11739330B2 (en) | 2017-09-22 | 2023-08-29 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
| US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
| WO2019102381A1 (en) | 2017-11-21 | 2019-05-31 | Casebia Therapeutics Llp | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| KR102907245B1 (ko) | 2018-03-14 | 2026-01-05 | 에디타스 메디신, 인코포레이티드 | 혈색소병증 치료를 위한 시스템 및 방법 |
| AU2019237541B2 (en) | 2018-03-23 | 2025-02-13 | Editas Medicine, Inc. | CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome |
| US20210017509A1 (en) * | 2018-03-23 | 2021-01-21 | The Trustees Of Columbia University In The City Of New York | Gene Editing for Autosomal Dominant Diseases |
| US12121563B2 (en) | 2018-04-09 | 2024-10-22 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3801640A4 (en) | 2018-05-30 | 2022-03-23 | The Regents Of The University Of California | GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED AGAMMAGLOBULINEMIA (XLA) |
| GB201809588D0 (en) * | 2018-06-12 | 2018-07-25 | Univ Bristol | Materials and methods for modulating intraocular and intracranial pressure |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| WO2020027982A1 (en) * | 2018-08-02 | 2020-02-06 | Editas Medicine, Inc. | Compositions and methods for treating cep290-associated disease |
| KR102103103B1 (ko) * | 2018-08-16 | 2020-04-21 | (주)라트바이오 | 인위적 뉴클레아제를 생산하는 형질전환 동물 및 형질전환 배아 |
| KR20210082460A (ko) | 2018-10-01 | 2021-07-05 | 울트라제닉스 파마수티컬 인코포레이티드 | 프로피온산혈증을 치료하기 위한 유전자 요법 |
| AU2019358806B2 (en) | 2018-10-08 | 2025-06-05 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in interneurons |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| AU2019385506A1 (en) * | 2018-11-21 | 2021-06-03 | Takeda Pharmaceutical Company Limited | Recombinant viral vectors and nucleic acids for producing the same |
| IT201800020230A1 (it) * | 2018-12-19 | 2020-06-19 | Univ Degli Studi Di Siena | Sistema CRISPR-Cas per la terapia genica. |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US20220047618A1 (en) * | 2019-02-28 | 2022-02-17 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| EP3942043A2 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| SG11202110613SA (en) * | 2019-03-27 | 2021-10-28 | Res Inst Nationwide Childrens Hospital | Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses |
| KR20210113393A (ko) * | 2019-03-28 | 2021-09-15 | 주식회사 툴젠 | 혈액응고인자 viii 유전자 역위 보정에 의한 혈우병 치료용 조성물 |
| CN114072497B (zh) * | 2019-04-15 | 2025-11-18 | 宾夕法尼亚州大学信托人 | 用于调控酶表达和使酶表达自失活的组合物以及用于调节酶的脱靶活性的方法 |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | 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 |
| CN111370055B (zh) * | 2020-03-05 | 2023-05-23 | 中南大学 | 内含子保留预测模型建立方法及其预测方法 |
| WO2021195218A1 (en) * | 2020-03-24 | 2021-09-30 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics |
| BR112022022603A2 (pt) | 2020-05-08 | 2023-01-17 | Broad Inst Inc | Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla |
| US12171813B2 (en) | 2021-02-05 | 2024-12-24 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| AU2022229489A1 (en) * | 2021-03-04 | 2023-08-31 | Research Institute At Nationwide Children's Hospital | Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications |
| US20220325298A1 (en) * | 2021-04-12 | 2022-10-13 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Crispr epigenetic therapeutics for pain management |
| JP7780172B2 (ja) * | 2021-07-30 | 2025-12-04 | 国立研究開発法人理化学研究所 | 筋萎縮性側索硬化症におけるｃｅｐ２９０遺伝子の変異 |
| CN114306277B (zh) * | 2021-11-02 | 2023-08-04 | 中国人民解放军空军军医大学 | 一种靶向psma的巨噬细胞膜包裹的仿生纳米药物载体及应用 |
| EP4223877A1 (en) | 2022-02-08 | 2023-08-09 | Eberhard Karls Universität Tübingen Medizinische Fakultät | System and method for editing genomic dna to modulate splicing |
| WO2025135916A1 (ko) * | 2023-12-22 | 2025-06-26 | 주식회사 툴젠 | 낮은 면역원성을 가지는 cas9 단백질 변이체 |
Family Cites Families (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| AU7979491A (en) | 1990-05-03 | 1991-11-27 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| US8802080B2 (en) | 2002-05-01 | 2014-08-12 | University Of Florida Research Foundation, Inc. | Raav expression systems for genetic modification of specific capsid proteins |
| US7261544B2 (en) | 2003-05-21 | 2007-08-28 | Genzyme Corporation | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2005056762A2 (en) * | 2003-12-05 | 2005-06-23 | University Of Iowa Research Foundation | Truncated cmv promoters and vectors containing same |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| WO2009105690A2 (en) | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| WO2009121536A1 (en) | 2008-04-02 | 2009-10-08 | Fondazione Telethon | Method of treating genetic disorders |
| KR101812813B1 (ko) | 2009-06-16 | 2017-12-27 | 젠자임 코포레이션 | 재조합 ａａｖ 벡터에 대한 개선된 정제 방법 |
| WO2012099540A1 (en) * | 2011-01-17 | 2012-07-26 | Agency For Science, Technology And Research | Cmv promoter variants |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CN105793425B (zh) * | 2013-06-17 | 2021-10-26 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| CN106459995B (zh) * | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| BR112016013547A2 (pt) | 2013-12-12 | 2017-10-03 | Broad Inst Inc | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
| CA2948728A1 (en) * | 2014-03-10 | 2015-09-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| RU2021132397A (ru) | 2015-05-16 | 2022-02-24 | Джензим Корпорейшн | Генное редактирование глубоких интронных мутаций |
| EP3310369B1 (en) * | 2015-06-19 | 2022-05-04 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| WO2017136335A1 (en) * | 2016-02-01 | 2017-08-10 | The Regents Of The University Of California | Self-inactivating endonuclease-encoding nucleic acids and methods of using the same |
-
2016
- 2016-04-15 RU RU2021132397A patent/RU2021132397A/ru unknown
- 2016-04-15 US US15/574,420 patent/US11896651B2/en active Active
- 2016-04-15 PL PL16718840.8T patent/PL3298134T3/pl unknown
- 2016-04-15 KR KR1020237033136A patent/KR20230142649A/ko active Pending
- 2016-04-15 CN CN202210293007.1A patent/CN114657180A/zh active Pending
- 2016-04-15 SG SG10201910750WA patent/SG10201910750WA/en unknown
- 2016-04-15 AU AU2016265255A patent/AU2016265255B2/en active Active
- 2016-04-15 EP EP16718840.8A patent/EP3298134B8/en active Active
- 2016-04-15 KR KR1020177036029A patent/KR102584873B1/ko active Active
- 2016-04-15 PT PT167188408T patent/PT3298134T/pt unknown
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