JP2024508696A5 - - Google Patents

Info

Publication number
JP2024508696A5
JP2024508696A5 JP2023548288A JP2023548288A JP2024508696A5 JP 2024508696 A5 JP2024508696 A5 JP 2024508696A5 JP 2023548288 A JP2023548288 A JP 2023548288A JP 2023548288 A JP2023548288 A JP 2023548288A JP 2024508696 A5 JP2024508696 A5 JP 2024508696A5
Authority
JP
Japan
Prior art keywords
rna
optionally
dna virus
sequence
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.)
Pending
Application number
JP2023548288A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024508696A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/CN2022/075366 external-priority patent/WO2022166954A1/en
Publication of JP2024508696A publication Critical patent/JP2024508696A/ja
Publication of JP2024508696A5 publication Critical patent/JP2024508696A5/ja
Pending legal-status Critical Current

Links

JP2023548288A 2021-02-07 2022-02-07 Rnaアデノ随伴ウイルス(raav)ベクター及びその用途 Pending JP2024508696A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2021075874 2021-02-07
CNPCT/CN2021/075874 2021-02-07
PCT/CN2022/075366 WO2022166954A1 (en) 2021-02-07 2022-02-07 Rna adeno-associated virus (raav) vector and uses thereof

Publications (2)

Publication Number Publication Date
JP2024508696A JP2024508696A (ja) 2024-02-28
JP2024508696A5 true JP2024508696A5 (enExample) 2025-02-18

Family

ID=74946911

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023548288A Pending JP2024508696A (ja) 2021-02-07 2022-02-07 Rnaアデノ随伴ウイルス(raav)ベクター及びその用途

Country Status (10)

Country Link
US (2) US12031148B2 (enExample)
EP (1) EP4288112A4 (enExample)
JP (1) JP2024508696A (enExample)
KR (1) KR20230142776A (enExample)
CN (1) CN115209924A (enExample)
AU (1) AU2022217349A1 (enExample)
BR (1) BR112023015769A2 (enExample)
CA (1) CA3207601A1 (enExample)
IL (1) IL305002A (enExample)
WO (1) WO2022166954A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116218904B (zh) * 2022-11-03 2025-04-15 国药中生生物技术研究院有限公司 一种可提高工程化细胞外泌体中特定核酸分子载量的方法及其应用
WO2024120528A1 (en) * 2022-12-08 2024-06-13 Huidagene Therapeutics (Singapore) Pte. Ltd. Improved system for producing rna-packaged aav particles
CN117778431B (zh) * 2023-12-04 2024-10-25 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
WO2025124549A1 (en) * 2023-12-13 2025-06-19 Huidagene Therapeutics (Singapore) Pte. Ltd. Improved system and method for producing recombinant dna-packaged aav particles
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法
WO2026095803A1 (en) * 2024-11-04 2026-05-07 Umc Utrecht Holding B.V. Sirna delivery with biological vectors

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
JP6738728B2 (ja) * 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのCRISPR−Cas系、ベクターおよび組成物の送達および使用
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP3957731A1 (en) * 2015-07-15 2022-02-23 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US11352647B2 (en) * 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3565608A4 (en) * 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES
JP7215716B2 (ja) * 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター
CN111328290A (zh) * 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
SG11201912024RA (en) * 2017-07-31 2020-02-27 Sigma Aldrich Co Llc Synthetic guide rna for crispr/cas activator systems
CN109055375B (zh) * 2018-07-26 2021-11-12 东南大学 一种crispr辅助反式增强子激活基因表达的方法及其应用
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
MX2023001164A (es) * 2020-07-29 2023-09-15 Univ America Catholic Diseño de virus artificiales a base de bacteriofagos para la remodelación del genoma humano.

Similar Documents

Publication Publication Date Title
JP2020019772A5 (enExample)
JP2024009857A5 (enExample)
JP2022508182A5 (enExample)
JP2021106619A5 (enExample)
JP2022512726A5 (enExample)
RU2018140499A (ru) Способы и композиции для лечения хореи гентингтона
JP2017510298A5 (enExample)
JP2007507223A5 (enExample)
JP2022551744A5 (enExample)
JP2021534814A5 (enExample)
JP2024508696A (ja) Rnaアデノ随伴ウイルス(raav)ベクター及びその用途
CN113897396B (zh) 一种用于在昆虫细胞中表达包含重叠开放阅读框的基因的表达盒及其应用
JP2025540074A (ja) 繰り返しエレメントの適切なパッケージングのためのアデノ随伴ウイルスベクター
JP2023541443A5 (enExample)
CN110770344B (zh) shRNA表达框、携带其的多核苷酸序列及其应用
JP2023537358A5 (enExample)
WO2024220722A1 (en) Compositions and methods for tissue- specific expression of gene therapies
JP2025513785A5 (enExample)
JP2022527126A5 (enExample)
RU2023121263A (ru) Вектор на основе рнк-содержащего аденоассоциированного вируса (raav) и пути его применения
CN111527201A (zh) 修饰的病毒载体及其制备和使用方法
JP2025509262A5 (enExample)
JP2025512709A5 (enExample)
JP2025515030A5 (enExample)
JP2022523320A5 (enExample)