JP2023525476A5 - - Google Patents

Info

Publication number
JP2023525476A5
JP2023525476A5 JP2022564157A JP2022564157A JP2023525476A5 JP 2023525476 A5 JP2023525476 A5 JP 2023525476A5 JP 2022564157 A JP2022564157 A JP 2022564157A JP 2022564157 A JP2022564157 A JP 2022564157A JP 2023525476 A5 JP2023525476 A5 JP 2023525476A5
Authority
JP
Japan
Prior art keywords
cns
synthetic
expression cassette
vector
seq
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
Application number
JP2022564157A
Other languages
English (en)
Japanese (ja)
Other versions
JP7807393B2 (ja
JP2023525476A (ja
Filing date
Publication date
Priority claimed from GBGB2005732.9A external-priority patent/GB202005732D0/en
Application filed filed Critical
Publication of JP2023525476A publication Critical patent/JP2023525476A/ja
Publication of JP2023525476A5 publication Critical patent/JP2023525476A5/ja
Priority to JP2026005291A priority Critical patent/JP2026082853A/ja
Application granted granted Critical
Publication of JP7807393B2 publication Critical patent/JP7807393B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022564157A 2020-04-20 2021-04-19 調節核酸配列 Active JP7807393B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026005291A JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2005732.9 2020-04-20
GBGB2005732.9A GB202005732D0 (en) 2020-04-20 2020-04-20 Regulatory nucleic acid sequences
PCT/GB2021/050939 WO2021214443A1 (en) 2020-04-20 2021-04-19 Regulatory nucleic acid sequences

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026005291A Division JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Publications (3)

Publication Number Publication Date
JP2023525476A JP2023525476A (ja) 2023-06-16
JP2023525476A5 true JP2023525476A5 (enExample) 2024-05-01
JP7807393B2 JP7807393B2 (ja) 2026-01-27

Family

ID=70860129

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022564157A Active JP7807393B2 (ja) 2020-04-20 2021-04-19 調節核酸配列
JP2026005291A Pending JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2026005291A Pending JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Country Status (8)

Country Link
US (1) US20230143758A1 (enExample)
EP (1) EP4139466A1 (enExample)
JP (2) JP7807393B2 (enExample)
CN (1) CN115702246B (enExample)
AU (1) AU2021260914A1 (enExample)
CA (1) CA3176038A1 (enExample)
GB (1) GB202005732D0 (enExample)
WO (1) WO2021214443A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023211606A1 (en) * 2022-01-27 2024-07-11 AskBio Inc. Compositions for treating neurological disease
AU2023226080A1 (en) * 2022-02-24 2024-10-03 Allen Institute Artificial expression constructs for modulating gene expression in dopaminergic neurons

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP0968284B1 (en) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral 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
JP2001517454A (ja) 1997-09-19 2001-10-09 ザ・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 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
ATE286138T1 (de) 1998-03-20 2005-01-15 Univ Pennsylvania Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
WO2000002053A2 (en) * 1998-07-03 2000-01-13 Innogenetics N.V. Differential diagnosis of neurodegeneration
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
JP2003501067A (ja) 1999-06-02 2003-01-14 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 ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
WO2001023001A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Rapid peg-modification
CN1247785C (zh) 2000-10-13 2006-03-29 希龙公司 巨细胞病毒内含子a片段
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
WO2005001103A2 (en) 2003-06-20 2005-01-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
EP1747277B1 (en) * 2004-04-14 2011-08-10 Agency for Science, Technology and Research Gene delivery to neuronal cells
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
IS8576A (is) * 2006-12-05 2007-01-15 Decode Genetics Genetic susceptibility variants of atrial fibrillation and stroke
ES2582200T3 (es) * 2007-09-12 2016-09-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Utilización de vectores víricos portadores del gen CYP46A1 para el tratamiento de la enfermedad de Alzheimer
WO2009074968A2 (en) * 2007-12-12 2009-06-18 Ecole Polytechnique Federale De Lausanne (Epfl) Method for predicting the efficacy of cancer therapy
US8383803B2 (en) * 2008-08-11 2013-02-26 The University Of British Columbia PITX3 expression promoters
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
DK2561073T3 (en) * 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
CN109536494A (zh) * 2017-10-31 2019-03-29 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒

Similar Documents

Publication Publication Date Title
JP2023078454A (ja) 調節性ポリヌクレオチド
CN112040988A (zh) 修饰的aav衣壳多肽用于治疗肌肉疾病
JP2024050553A (ja) 不整脈源性右心室心筋症における電気的機能及び心臓機能並びに心構造を回復するための遺伝子療法戦略
JP2026021360A5 (enExample)
JP2023509118A5 (enExample)
IL320371A (en) Gene therapy for patients with fanconi anemia
CN114432332A (zh) circUTRN在制备治疗心力衰竭药物中的应用、重组载体和治疗心力衰竭的药物
JP2025170345A5 (enExample)
CN118126156A (zh) 一种多肽及其用途
CN108888753B (zh) 酸性糖蛋白orm在制备治疗心力衰竭药物中的应用
JP2024524270A5 (enExample)
CN114736924B (zh) 异位联合过表达Atoh1和Ikzf2再生耳蜗外毛细胞及其应用
WO2020223933A1 (zh) 高效感染支持细胞的aav突变体
EP4475864A1 (en) Methods for treating stroke using a stanniocalcin 2 (stc2) pharmaceutical composition
CN116574798A (zh) piR-mmu-57256903在制备防治抗癌药物所致心脏疾病的药物中的应用
US20240026355A1 (en) Compositions for and methods of enhancing tissue regeneration
CN111909246B (zh) 高效感染支持细胞的aav突变体
JP2024519955A5 (enExample)
WO2022135278A1 (zh) Azd3965药物的新用途
JP2023540545A5 (enExample)
US20250360171A1 (en) Chemogenetically gated ion channels and use thereof
WO2025018299A1 (ja) 血管平滑筋細胞指向カプシド、アデノ随伴ウイルスベクター、医薬組成物、治療方法、核酸導入方法、及びカプシド製造方法
Notkin et al. Comparative study of metrazol treatment and control observations of schizophrenia
CA2629304C (en) Combination of glycoisoforms for the treatment or prevention of septicemia, transgenic cell line that produces erythropoietin glycoisoforms, pharmaceutical composition comprising said combination
WO2025058068A1 (ja) 心筋再生、心筋線維化の抑制または心疾患の処置用の医薬組成物