JPWO2020247882A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020247882A5 JPWO2020247882A5 JP2021572556A JP2021572556A JPWO2020247882A5 JP WO2020247882 A5 JPWO2020247882 A5 JP WO2020247882A5 JP 2021572556 A JP2021572556 A JP 2021572556A JP 2021572556 A JP2021572556 A JP 2021572556A JP WO2020247882 A5 JPWO2020247882 A5 JP WO2020247882A5
- Authority
- JP
- Japan
- Prior art keywords
- improved
- grna
- sequence
- seq
- protein
- 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 (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025147107A JP2025183286A (ja) | 2019-06-07 | 2025-09-04 | 操作されたｃａｓｘシステム |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962858750P | 2019-06-07 | 2019-06-07 | |
| US62/858,750 | 2019-06-07 | ||
| US201962944892P | 2019-12-06 | 2019-12-06 | |
| US62/944,892 | 2019-12-06 | ||
| US202063030838P | 2020-05-27 | 2020-05-27 | |
| US63/030,838 | 2020-05-27 | ||
| PCT/US2020/036505 WO2020247882A1 (en) | 2019-06-07 | 2020-06-05 | Engineered casx systems |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025147107A Division JP2025183286A (ja) | 2019-06-07 | 2025-09-04 | 操作されたｃａｓｘシステム |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022534809A JP2022534809A (ja) | 2022-08-03 |
| JP2022534809A5 JP2022534809A5 (https=) | 2023-06-12 |
| JPWO2020247882A5 true JPWO2020247882A5 (https=) | 2023-06-12 |
| JP7744011B2 JP7744011B2 (ja) | 2025-09-25 |
Family
ID=71899867
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021572556A Active JP7744011B2 (ja) | 2019-06-07 | 2020-06-05 | 操作されたｃａｓｘシステム |
| JP2025147107A Pending JP2025183286A (ja) | 2019-06-07 | 2025-09-04 | 操作されたｃａｓｘシステム |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025147107A Pending JP2025183286A (ja) | 2019-06-07 | 2025-09-04 | 操作されたｃａｓｘシステム |
Country Status (17)
| Country | Link |
|---|---|
| US (5) | US11560555B2 (https=) |
| EP (1) | EP3980533A1 (https=) |
| JP (2) | JP7744011B2 (https=) |
| KR (1) | KR20220032050A (https=) |
| CN (1) | CN114375334B (https=) |
| AU (1) | AU2020289591A1 (https=) |
| BR (1) | BR112021024288A2 (https=) |
| CA (1) | CA3142883A1 (https=) |
| CL (1) | CL2021003233A1 (https=) |
| GB (1) | GB2600274B (https=) |
| IL (1) | IL288738A (https=) |
| MX (1) | MX2021015058A (https=) |
| PE (1) | PE20220256A1 (https=) |
| PH (1) | PH12021553076A1 (https=) |
| SG (1) | SG11202113253SA (https=) |
| TW (1) | TW202113074A (https=) |
| WO (1) | WO2020247882A1 (https=) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12275964B2 (en) | 2018-08-22 | 2025-04-15 | The Regents Of The University Of California | Variant type V CRISPR/Cas effector polypeptides and methods of use thereof |
| PH12021553076A1 (en) | 2019-06-07 | 2023-08-14 | Scribe Therapeutics Inc | Engineered casx systems |
| CA3153700A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
| US20220348925A1 (en) | 2019-09-09 | 2022-11-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of sod1 |
| CA3159316A1 (en) | 2019-12-06 | 2021-06-10 | Benjamin OAKES | Compositions and methods for the targeting of rhodopsin |
| AU2021206270A1 (en) * | 2020-01-10 | 2022-07-21 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of PCSK9 |
| BR112022018673A2 (pt) | 2020-03-18 | 2022-12-27 | Scribe Therapeutics Inc | Composições e métodos para o direcionamento de c9orf72 |
| GB202010075D0 (en) * | 2020-07-01 | 2020-08-12 | Imp College Innovations Ltd | Therapeutic nucleic acids, peptides and uses |
| WO2022119957A1 (en) * | 2020-12-02 | 2022-06-09 | The Regents Of The University Of California | Crispr-cas effector polypeptides and methods of use thereof |
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| US20240100185A1 (en) | 2020-12-03 | 2024-03-28 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of ptbp1 |
| EP4259791A1 (en) | 2020-12-09 | 2023-10-18 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2022192381A1 (en) * | 2021-03-09 | 2022-09-15 | Arbor Biotechnologies, Inc. | Compositions comprising a variant polypeptide and uses thereof |
| IL308806A (en) | 2021-06-01 | 2024-01-01 | Arbor Biotechnologies Inc | Gene editing systems including nuclease crisper and their uses |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| JP2024534523A (ja) * | 2021-09-21 | 2024-09-20 | スクライブ・セラピューティクス・インコーポレイテッド | 操作されたｃａｓｘリプレッサー系 |
| IL311611A (en) | 2021-09-23 | 2024-05-01 | Scribe Therapeutics Inc | Self-inactivating gene-editing vectors |
| CN116497002A (zh) * | 2022-01-19 | 2023-07-28 | 中国科学院动物研究所 | 工程化的CasX核酸酶、效应蛋白及其用途 |
| WO2023235818A2 (en) | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| WO2023240076A1 (en) * | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| WO2023240074A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| US20250361525A1 (en) | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2023240157A2 (en) | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
| WO2024081711A2 (en) * | 2022-10-11 | 2024-04-18 | The Broad Institute, Inc. | Reprogramable tnpb polypeptides and use thereof |
| WO2024173645A1 (en) | 2023-02-15 | 2024-08-22 | Arbor Biotechnologies, Inc. | Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript |
| WO2024192291A1 (en) | 2023-03-15 | 2024-09-19 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| TW202444910A (zh) | 2023-03-29 | 2024-11-16 | 美商斯奎柏治療公司 | 編碼casx之信使rna |
| TW202444906A (zh) | 2023-03-29 | 2024-11-16 | 美商斯奎柏治療公司 | 用於靶向pcsk9之組合物及方法 |
| WO2024206676A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of lpa |
| WO2024206565A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Repressor fusion protein systems |
| CN116926043B (zh) * | 2023-04-28 | 2024-09-06 | 新乡医学院第三附属医院 | 一种dCasX蛋白核酸酶、重组载体及其应用 |
| WO2024228975A2 (en) * | 2023-05-02 | 2024-11-07 | Inari Agriculture Technology, Inc. | Ribozyme sequences for processing rna |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
| AU2024302819A1 (en) * | 2023-06-16 | 2026-01-22 | Hiroshima University | Zinc finger protein, zinc finger nuclease, vector, and genome editing method using said substances |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025072293A1 (en) | 2023-09-27 | 2025-04-03 | Scribe Therapeutics Inc. | Optimized mrnas encoding casx proteins |
| CN117296799B (zh) * | 2023-11-28 | 2024-02-02 | 四川省医学科学院·四川省人民医院 | 一种视网膜色素变性疾病模型的构建方法及其应用 |
| WO2025171210A1 (en) | 2024-02-09 | 2025-08-14 | Arbor Biotechnologies, Inc. | Compositions and methods for gene editing via homology-mediated end joining |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025240940A1 (en) | 2024-05-17 | 2025-11-20 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of apolipoprotein c3 |
| WO2026015832A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Reverse transcriptases and gene editing systems comprising such |
| WO2026015829A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Small reverse transcriptases and gene editing systems comprising such |
| CN120424908B (zh) * | 2025-07-10 | 2025-09-12 | 南京农业大学 | 一种基于LAMP与CRISPR/Cas的检测方法和应用 |
Family Cites Families (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5208036A (en) | 1985-01-07 | 1993-05-04 | 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 |
| US5223409A (en) | 1988-09-02 | 1993-06-29 | Protein Engineering Corp. | Directed evolution of novel binding proteins |
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US5279833A (en) | 1990-04-04 | 1994-01-18 | Yale University | Liposomal transfection of nucleic acids into animal cells |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| US5412087A (en) | 1992-04-24 | 1995-05-02 | Affymax Technologies N.V. | Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| ATE219660T1 (de) | 1994-09-30 | 2002-07-15 | Inex Pharmaceuticals Corp | Mittel zum einbringen polyanionischer materialien in zellen |
| US5695937A (en) | 1995-09-12 | 1997-12-09 | The Johns Hopkins University School Of Medicine | Method for serial analysis of gene expression |
| US7745651B2 (en) | 2004-06-07 | 2010-06-29 | Protiva Biotherapeutics, Inc. | Cationic lipids and methods of use |
| JP5042863B2 (ja) | 2005-02-14 | 2012-10-03 | サーナ・セラピューティクス・インコーポレイテッド | 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法 |
| WO2009086558A1 (en) | 2008-01-02 | 2009-07-09 | Tekmira Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| AU2009238175C1 (en) | 2008-04-15 | 2023-11-30 | Arbutus Biopharma Corporation | Novel lipid formulations for nucleic acid delivery |
| DK2356270T3 (da) | 2008-11-07 | 2016-12-12 | Fabrus Llc | Kombinatoriske antistofbiblioteker og anvendelser deraf |
| WO2010075303A1 (en) | 2008-12-23 | 2010-07-01 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same |
| US20110071208A1 (en) | 2009-06-05 | 2011-03-24 | Protiva Biotherapeutics, Inc. | Lipid encapsulated dicer-substrate interfering rna |
| ES2613498T3 (es) | 2009-07-01 | 2017-05-24 | Protiva Biotherapeutics Inc. | Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| SMT202200502T1 (it) | 2014-06-25 | 2023-01-13 | Acuitas Therapeutics Inc | Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici |
| US10900034B2 (en) | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| US11219634B2 (en) | 2015-01-21 | 2022-01-11 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| EP4159856A1 (en) | 2015-06-18 | 2023-04-05 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| LT3313829T (lt) | 2015-06-29 | 2024-08-12 | Acuitas Therapeutics Inc. | Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui |
| CN109153980B (zh) | 2015-10-22 | 2023-04-14 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
| IL307179A (en) | 2015-10-28 | 2023-11-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| WO2017083722A1 (en) | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
| WO2017106569A1 (en) | 2015-12-18 | 2017-06-22 | The Regents Of The University Of California | Modified site-directed modifying polypeptides and methods of use thereof |
| US9896696B2 (en) | 2016-02-15 | 2018-02-20 | Benson Hill Biosystems, Inc. | Compositions and methods for modifying genomes |
| WO2017212008A1 (en) | 2016-06-09 | 2017-12-14 | Curevac Ag | Hybrid carriers for nucleic acid cargo |
| WO2017212007A1 (en) | 2016-06-09 | 2017-12-14 | Curevac Ag | Cationic carriers for nucleic acid delivery |
| US10017760B2 (en) | 2016-06-24 | 2018-07-10 | Inscripta, Inc. | Methods for generating barcoded combinatorial libraries |
| US12431216B2 (en) | 2016-08-17 | 2025-09-30 | Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| CA3038960A1 (en) | 2016-09-30 | 2018-04-05 | The Regents Of The University Of California | Rna-guided nucleic acid modifying enzymes and methods of use thereof |
| US9982267B2 (en) | 2016-10-12 | 2018-05-29 | Feldan Bio Inc. | Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same |
| IL267470B2 (en) | 2017-01-10 | 2023-10-01 | F O R E Biotherapeutics Ltd | Methods for in vitro site-directed mutagenesis using gene editing technologies |
| WO2018152418A1 (en) | 2017-02-17 | 2018-08-23 | Temple University - Of The Commonwealth System Of Higher Education | Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5 |
| AU2018240515B2 (en) | 2017-03-24 | 2024-07-25 | CureVac SE | Nucleic acids encoding CRISPR-associated proteins and uses thereof |
| WO2018195555A1 (en) | 2017-04-21 | 2018-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors |
| US11578334B2 (en) | 2017-10-25 | 2023-02-14 | Monsanto Technology Llc | Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes |
| US10253365B1 (en) | 2017-11-22 | 2019-04-09 | The Regents Of The University Of California | Type V CRISPR/Cas effector proteins for cleaving ssDNAs and detecting target DNAs |
| CN111770758A (zh) | 2018-02-27 | 2020-10-13 | 北卡罗来纳-查佩尔山大学 | 用于治疗Angelman综合征的方法和组合物 |
| US20200224160A1 (en) | 2018-02-27 | 2020-07-16 | Sorrento Therapeutics, Inc. | Process for dna integration using rna-guided endonucleases |
| US12522811B2 (en) | 2018-05-01 | 2026-01-13 | The Children's Medical Center Corporation | Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9 |
| WO2020010083A1 (en) | 2018-07-05 | 2020-01-09 | The Regents Of The University Of California | Compositions and methods for delivery of rna to a cell |
| WO2020023529A1 (en) | 2018-07-24 | 2020-01-30 | The Regents Of The University Of California | Rna-guided nucleic acid modifying enzymes and methods of use thereof |
| US12275964B2 (en) | 2018-08-22 | 2025-04-15 | The Regents Of The University Of California | Variant type V CRISPR/Cas effector polypeptides and methods of use thereof |
| PH12021553076A1 (en) | 2019-06-07 | 2023-08-14 | Scribe Therapeutics Inc | Engineered casx systems |
| WO2020247883A2 (en) | 2019-06-07 | 2020-12-10 | Scribe Therapeutics Inc. | Deep mutational evolution of biomolecules |
| US12454685B2 (en) | 2019-07-08 | 2025-10-28 | The Regents Of The University Of California | Variant type V CRISPR/Cas effector polypeptides and methods of use thereof |
| US12371695B2 (en) | 2019-08-02 | 2025-07-29 | Monsanto Technology Llc | Methods and compositions to promote targeted genome modifications using HUH endonucleases |
| CA3153700A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
| US20220348925A1 (en) | 2019-09-09 | 2022-11-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of sod1 |
| IL292571A (en) | 2019-10-28 | 2022-06-01 | Targetgene Biotechnologies Ltd | Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation |
| AU2020398658A1 (en) | 2019-12-06 | 2022-07-07 | Scribe Therapeutics Inc. | Particle delivery systems |
| CA3159316A1 (en) | 2019-12-06 | 2021-06-10 | Benjamin OAKES | Compositions and methods for the targeting of rhodopsin |
| EP4069846A1 (en) | 2019-12-07 | 2022-10-12 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of htt |
| AU2021206270A1 (en) | 2020-01-10 | 2022-07-21 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of PCSK9 |
| BR112022018673A2 (pt) | 2020-03-18 | 2022-12-27 | Scribe Therapeutics Inc | Composições e métodos para o direcionamento de c9orf72 |
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| JP2024534523A (ja) | 2021-09-21 | 2024-09-20 | スクライブ・セラピューティクス・インコーポレイテッド | 操作されたｃａｓｘリプレッサー系 |
| IL311611A (en) | 2021-09-23 | 2024-05-01 | Scribe Therapeutics Inc | Self-inactivating gene-editing vectors |
| WO2023235818A2 (en) | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
-
2020
- 2020-06-05 PH PH1/2021/553076A patent/PH12021553076A1/en unknown
- 2020-06-05 CN CN202080056141.9A patent/CN114375334B/zh active Active
- 2020-06-05 KR KR1020227000734A patent/KR20220032050A/ko active Pending
- 2020-06-05 EP EP20750444.0A patent/EP3980533A1/en active Pending
- 2020-06-05 BR BR112021024288A patent/BR112021024288A2/pt unknown
- 2020-06-05 TW TW109119111A patent/TW202113074A/zh unknown
- 2020-06-05 AU AU2020289591A patent/AU2020289591A1/en active Pending
- 2020-06-05 CA CA3142883A patent/CA3142883A1/en active Pending
- 2020-06-05 SG SG11202113253SA patent/SG11202113253SA/en unknown
- 2020-06-05 MX MX2021015058A patent/MX2021015058A/es unknown
- 2020-06-05 GB GB2200132.5A patent/GB2600274B/en active Active
- 2020-06-05 JP JP2021572556A patent/JP7744011B2/ja active Active
- 2020-06-05 WO PCT/US2020/036505 patent/WO2020247882A1/en not_active Ceased
- 2020-06-05 PE PE2021002050A patent/PE20220256A1/es unknown
-
2021
- 2021-11-23 US US17/533,997 patent/US11560555B2/en active Active
- 2021-12-03 US US17/542,219 patent/US20220220508A1/en active Pending
- 2021-12-06 IL IL288738A patent/IL288738A/en unknown
- 2021-12-06 CL CL2021003233A patent/CL2021003233A1/es unknown
-
2022
- 2022-11-22 US US18/058,251 patent/US12084692B2/en active Active
-
2024
- 2024-07-19 US US18/778,393 patent/US20250043262A1/en active Pending
- 2024-10-08 US US18/909,093 patent/US20250136962A1/en active Pending
-
2025
- 2025-09-04 JP JP2025147107A patent/JP2025183286A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2020247882A5 (https=) | ||
| US11965174B2 (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| JP7651190B2 (ja) | 遺伝子操作ｒｎａを用いた内因性ａｄａｒによる標的化ｒｎａ編集 | |
| JP7374119B2 (ja) | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 | |
| GB2600274A (en) | Engineered CasX systems | |
| JP7004328B2 (ja) | 高形質導入効率ｒＡＡＶベクター、組成物、および使用方法 | |
| AU2016358172B2 (en) | Materials and methods for treatment of titin-based myopathies and other titinopaties | |
| JP2024028643A5 (https=) | ||
| JP2024161365A (ja) | Ａａｖキャプシドの転写依存性定方向進化を使用するための方法 | |
| CN112512595A (zh) | 心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复 | |
| JP7481755B2 (ja) | 組換えパルボウイルスベクターならびにその作製および使用方法 | |
| JP2022541070A (ja) | 調節可能な発現系 | |
| WO2025061173A1 (zh) | 一种融合蛋白及其应用 | |
| CN111718420B (zh) | 一种用于基因治疗的融合蛋白及其应用 | |
| JPWO2021188729A5 (https=) | ||
| JP2023507174A (ja) | Ｄｍｄ変異の修正のための方法及び組成物 | |
| JPWO2022120094A5 (https=) | ||
| CN120818522B (zh) | 一种基于itr-增强子驱动核酸表达的调控元件组合及其应用 | |
| CN105358690B (zh) | 启动子组合物 | |
| CN118955653A (zh) | 腺相关病毒衣壳蛋白的突变体、编码其的核酸及应用 | |
| HK40049414A (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| HK1240977B (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| HK1240977A1 (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |