JP2023509118A5 - - Google Patents

Info

Publication number
JP2023509118A5
JP2023509118A5 JP2022539145A JP2022539145A JP2023509118A5 JP 2023509118 A5 JP2023509118 A5 JP 2023509118A5 JP 2022539145 A JP2022539145 A JP 2022539145A JP 2022539145 A JP2022539145 A JP 2022539145A JP 2023509118 A5 JP2023509118 A5 JP 2023509118A5
Authority
JP
Japan
Prior art keywords
seq
crm
functional variant
sequence number
cre0035
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
JP2022539145A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023509118A (ja
Filing date
Publication date
Priority claimed from GBGB1919269.9A external-priority patent/GB201919269D0/en
Priority claimed from GBGB2012192.7A external-priority patent/GB202012192D0/en
Application filed filed Critical
Priority claimed from PCT/GB2020/053371 external-priority patent/WO2021130503A1/en
Publication of JP2023509118A publication Critical patent/JP2023509118A/ja
Publication of JP2023509118A5 publication Critical patent/JP2023509118A5/ja
Pending legal-status Critical Current

Links

JP2022539145A 2019-12-24 2020-12-24 調節性核酸配列 Pending JP2023509118A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1919269.9 2019-12-24
GBGB1919269.9A GB201919269D0 (en) 2019-12-24 2019-12-24 Regulatory nucleic acid sequences
GB2012192.7 2020-08-05
GBGB2012192.7A GB202012192D0 (en) 2020-08-05 2020-08-05 Regulatory nucleic acid sequences
PCT/GB2020/053371 WO2021130503A1 (en) 2019-12-24 2020-12-24 Regulatory nucleic acid sequences

Publications (2)

Publication Number Publication Date
JP2023509118A JP2023509118A (ja) 2023-03-07
JP2023509118A5 true JP2023509118A5 (enExample) 2024-01-05

Family

ID=74130280

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022539145A Pending JP2023509118A (ja) 2019-12-24 2020-12-24 調節性核酸配列

Country Status (10)

Country Link
US (1) US20230233710A1 (enExample)
EP (1) EP4081643A1 (enExample)
JP (1) JP2023509118A (enExample)
KR (1) KR20220119703A (enExample)
CN (1) CN115151646A (enExample)
AU (1) AU2020412375A1 (enExample)
CA (1) CA3161735A1 (enExample)
IL (1) IL294238A (enExample)
WO (1) WO2021130503A1 (enExample)
ZA (1) ZA202207849B (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
CN116406297A (zh) * 2020-08-05 2023-07-07 阿斯克肋匹奥生物制药公司 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法
CA3221707A1 (en) * 2021-06-23 2022-12-29 Jorge Omar YANEZ-CUNA Regulatory nucleic acid sequences
JP2025512574A (ja) * 2022-04-22 2025-04-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー AAVベクターを使用するB細胞リンパ腫2関連Anthanogene 3 (BAG3)遺伝子治療
CN115948403B (zh) * 2022-12-30 2023-09-15 广州派真生物技术有限公司 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用
CN122396772A (zh) * 2023-12-18 2026-07-14 豪夫迈·罗氏有限公司 骨骼肌特异性启动子

Family Cites Families (16)

* 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
CN1247785C (zh) 2000-10-13 2006-03-29 希龙公司 巨细胞病毒内含子a片段
US20030074679A1 (en) * 2000-12-12 2003-04-17 Schwartz Robert J. Administration of nucleic acid sequence to female animal to enhance growth in offspring
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
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
EP2494058B1 (en) * 2009-10-29 2017-04-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
CN103074373A (zh) * 2011-10-26 2013-05-01 南京医科大学 肌肉组织特异性表达Follistatin以提高牛瘦肉率的转基因载体
CN102747082B (zh) * 2011-12-29 2014-06-18 华中农业大学 猪肌肉特异性itgb1bp2启动子及其应用
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2017021359A1 (en) * 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
CN105063079A (zh) * 2015-08-15 2015-11-18 内蒙古大学 一种三顺反子肌肉特异双向共表达基因转移体及制备方法
US20170253927A1 (en) * 2016-03-01 2017-09-07 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure
CN108018311A (zh) * 2016-11-04 2018-05-11 中国科学院上海生命科学研究院 通过基因编辑特异靶向肌肉组织mstn治疗恶病质
US11338045B2 (en) * 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy

Similar Documents

Publication Publication Date Title
Colella et al. Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients
Sandig et al. Optimization of the helper-dependent adenovirus system for production and potency in vivo
JP7415042B2 (ja) プラコフィリン2遺伝子治療の方法および組成物
Carelli et al. Mitochondria: biogenesis and mitophagy balance in segregation and clonal expansion of mitochondrial DNA mutations
JP2023513749A5 (enExample)
Savio-Galimberti et al. Cardiac arrhythmias related to sodium channel dysfunction
Grisorio et al. Gene therapy for cardiac diseases: methods, challenges, and future directions
JP2021510301A5 (enExample)
JP2024511163A5 (enExample)
US11872290B2 (en) Bicistronic AAV vector for RNA interference in ALS
JP2022551739A5 (enExample)
JP7466855B2 (ja) フリードライヒ運動失調症の治療用のベクター
JP2022507828A5 (enExample)
AU2010311376A1 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
JP2024545183A5 (enExample)
KR101860452B1 (ko) 간세포 성장인자의 둘 이상의 이형체를 이용한 근위축성 측삭 경화증 예방 또는 치료용 조성물
Tulalamba et al. Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors
Jane et al. Understanding fetal globin gene expression: a step towards effective HbF reactivation in haemoglobinopathies.
Hu et al. Long-term amelioration of an early-onset familial atrial fibrillation model with AAV-mediated in vivo gene therapy
WO2019162356A1 (en) Prevention or treatment of cardiac arrhytmia and sudden cardiac death
JP2023525476A5 (enExample)
CN107073078B (zh) 利用肝细胞生长因子和基质细胞衍生因子1α的用于预防或治疗外周动脉疾病的组合物
JP2024524270A5 (enExample)
JPH11318467A (ja) 短縮型ジストロフィン
JP2020535203A5 (enExample)