JP2018516978A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
Links
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 JP2024073536A (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 (cg-RX-API-DMAC7.html) | 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 Pending JP2024073536A (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 Pending JP2024073536A (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 (16)
Families Citing this family (76)
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| EP3750907A3 (en) | 2014-03-18 | 2021-04-28 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| US11242525B2 (en) | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| WO2015153780A1 (en) | 2014-04-02 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| EP3521437B1 (en) | 2014-11-21 | 2025-01-15 | Regeneron Pharmaceuticals, Inc. | Methods for targeted genetic modification using paired guide rnas |
| CA2982966C (en) | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| KR102584873B1 (ko) * | 2015-05-16 | 2023-10-10 | 젠자임 코포레이션 | 심부 인트론 돌연변이의 유전자 편집 |
| WO2016205825A1 (en) * | 2015-06-19 | 2016-12-22 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| EP3433364A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| NZ747857A (en) | 2016-05-20 | 2023-01-27 | Regeneron Pharma | Methods for breaking immunological tolerance using multiple guide rnas |
| BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | 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. |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| JP7272952B2 (ja) | 2016-12-05 | 2023-05-12 | エディタス・メディシン、インコーポレイテッド | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| CA3059213A1 (en) * | 2017-05-09 | 2018-11-15 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| 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 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CN118006607A (zh) | 2017-09-22 | 2024-05-10 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| US20190153440A1 (en) | 2017-11-21 | 2019-05-23 | Casebia Therapeutics Llp | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019178426A1 (en) | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| CA3094828A1 (en) * | 2018-03-23 | 2019-09-26 | Massachusetts Eye And Ear Infirmary | Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome |
| EP3768327A4 (en) * | 2018-03-23 | 2022-04-13 | The Trustees of Columbia University in the City of New York | GENE EDITING FOR AUTOSOMAL DOMINANT DISEASES |
| JP7472036B2 (ja) | 2018-04-09 | 2024-04-22 | アレン インスティテュート | 抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2019232251A1 (en) * | 2018-05-30 | 2019-12-05 | The Regents Of The University Of California | Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim) |
| GB201809588D0 (en) * | 2018-06-12 | 2018-07-25 | Univ Bristol | Materials and methods for modulating intraocular and intracranial pressure |
| 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 |
| CA3113975A1 (en) | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| JP7695192B2 (ja) | 2018-10-08 | 2025-06-18 | アレン インスティテュート | 介在ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物 |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| SG11202105326WA (en) * | 2018-11-21 | 2021-06-29 | Stridebio Inc | 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 |
| JP2022522756A (ja) * | 2019-02-28 | 2022-04-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| BR112021019180A2 (pt) * | 2019-03-27 | 2022-03-03 | Res Inst Nationwide Childrens Hospital | Geração de células nk primárias de receptor de antígeno quimérico (car) para imunoterapia de câncer usando uma combinação de vírus cas9/rnp e aav |
| WO2020197330A1 (ko) * | 2019-03-28 | 2020-10-01 | 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 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | 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 |
| CN115667531A (zh) * | 2020-03-24 | 2023-01-31 | 世代生物公司 | 非病毒DNA载体和其用于表达戈谢(Gaucher)治疗剂的用途 |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| EP4288541A1 (en) | 2021-02-05 | 2023-12-13 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2022187571A1 (en) * | 2021-03-04 | 2022-09-09 | 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | 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 |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | 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 instrastrand base pairs |
| CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| 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 |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| ATE405295T1 (de) | 2002-05-01 | 2008-09-15 | Univ Florida | Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| US7407801B2 (en) * | 2003-12-05 | 2008-08-05 | 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 |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20100081707A1 (en) | 2008-02-21 | 2010-04-01 | Ali Robin R | 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 |
| SG10201403290SA (en) | 2009-06-16 | 2014-10-30 | Genzyme Corp | Improved methods for purification of recombinant aav vectors |
| WO2012099540A1 (en) * | 2011-01-17 | 2012-07-26 | Agency For Science, Technology And Research | Cmv promoter variants |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| 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 |
| CN106062197A (zh) * | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
| CN111218447B (zh) * | 2013-11-07 | 2024-10-11 | 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 |
| 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 |
| US9938521B2 (en) * | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| KR102584873B1 (ko) * | 2015-05-16 | 2023-10-10 | 젠자임 코포레이션 | 심부 인트론 돌연변이의 유전자 편집 |
| WO2016205825A1 (en) * | 2015-06-19 | 2016-12-22 | 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 KR KR1020177036029A patent/KR102584873B1/ko active Active
- 2016-04-15 US US15/574,420 patent/US11896651B2/en active Active
- 2016-04-15 IL IL296365A patent/IL296365A/en unknown
- 2016-04-15 MX MX2017014665A patent/MX2017014665A/es unknown
- 2016-04-15 ES ES16718840T patent/ES2952598T3/es active Active
- 2016-04-15 SG SG10201910750WA patent/SG10201910750WA/en unknown
- 2016-04-15 KR KR1020237033136A patent/KR20230142649A/ko active Pending
- 2016-04-15 CA CA2986021A patent/CA2986021A1/en active Pending
- 2016-04-15 CN CN201680041691.7A patent/CN107849547B/zh active Active
- 2016-04-15 SG SG10201912907PA patent/SG10201912907PA/en unknown
- 2016-04-15 EP EP16718840.8A patent/EP3298134B8/en active Active
- 2016-04-15 RU RU2021132397A patent/RU2021132397A/ru unknown
- 2016-04-15 WO PCT/US2016/027987 patent/WO2016186772A2/en not_active Ceased
- 2016-04-15 JP JP2018511592A patent/JP6985250B2/ja active Active
- 2016-04-15 PT PT167188408T patent/PT3298134T/pt unknown
- 2016-04-15 AU AU2016265255A patent/AU2016265255B2/en active Active
- 2016-04-15 PL PL16718840.8T patent/PL3298134T3/pl unknown
- 2016-04-15 BR BR112017024514-0A patent/BR112017024514A2/pt active Search and Examination
- 2016-04-15 CN CN202210293007.1A patent/CN114657180A/zh active Pending
- 2016-04-15 CN CN202410714061.8A patent/CN118879692A/zh active Pending
- 2016-04-15 RU RU2017143997A patent/RU2759335C2/ru active
-
2017
- 2017-11-14 IL IL255654A patent/IL255654B2/en unknown
- 2017-11-15 MX MX2023006046A patent/MX2023006046A/es unknown
-
2021
- 2021-11-24 JP JP2021189812A patent/JP2022031769A/ja active Pending
-
2022
- 2022-06-16 AU AU2022204199A patent/AU2022204199B2/en active Active
-
2023
- 2023-12-28 US US18/399,364 patent/US20240197837A1/en active Pending
-
2024
- 2024-03-13 JP JP2024038537A patent/JP2024073536A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018516978A5 (cg-RX-API-DMAC7.html) | ||
| RU2017143997A (ru) | Генное редактирование глубоких интронных мутаций | |
| JP7561788B2 (ja) | 臨床使用に適した無血清懸濁細胞培養システムにおいて組換えアデノ随伴ウイルス(aav)ベクターを産生するスケーラブルな方法 | |
| CN105579465B (zh) | 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途 | |
| JP2020500541A5 (cg-RX-API-DMAC7.html) | ||
| JP2024099770A5 (cg-RX-API-DMAC7.html) | ||
| IL292999A (en) | Compositions and methods of treating amyotrophic lateral sclerosis (als) | |
| US11814642B2 (en) | Manufacturing and use of recombinant AAV vectors | |
| CN116194152A (zh) | 用于改进封闭端DNA(ceDNA)的产生的经修饰的杆状病毒系统 | |
| JP2020519284A5 (cg-RX-API-DMAC7.html) | ||
| JPWO2020082046A5 (cg-RX-API-DMAC7.html) | ||
| JPWO2019152609A5 (cg-RX-API-DMAC7.html) | ||
| JP2020509786A5 (cg-RX-API-DMAC7.html) | ||
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| IL292605A (en) | CRISPR and AAV strategies for the treatment of x-linked childhood retinoschisis | |
| JPWO2020214613A5 (cg-RX-API-DMAC7.html) | ||
| US20220177529A1 (en) | Fusion protein for enhancing gene editing and use thereof | |
| JPWO2020214609A5 (cg-RX-API-DMAC7.html) | ||
| Feng et al. | A 16 bp Rep binding element is sufficient for mediating Rep-dependent integration into AAVS1 | |
| US20250019667A1 (en) | Adenoviral helper plasmid | |
| WO2018204626A1 (en) | Regulatable gene editing compositions and methods | |
| JPWO2020082047A5 (cg-RX-API-DMAC7.html) | ||
| JPWO2020028816A5 (cg-RX-API-DMAC7.html) | ||
| CN115605266A (zh) | Dysferlin双载体的基因疗法 | |
| US20210261982A1 (en) | Raav-mediated nuclease-associated vector integration (raav-navi) |