JP2013509168A5 - - Google Patents

Download PDF

Info

Publication number
JP2013509168A5
JP2013509168A5 JP2012535849A JP2012535849A JP2013509168A5 JP 2013509168 A5 JP2013509168 A5 JP 2013509168A5 JP 2012535849 A JP2012535849 A JP 2012535849A JP 2012535849 A JP2012535849 A JP 2012535849A JP 2013509168 A5 JP2013509168 A5 JP 2013509168A5
Authority
JP
Japan
Prior art keywords
nucleic acid
expression cassette
vector
seq
acid expression
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
JP2012535849A
Other languages
English (en)
Japanese (ja)
Other versions
JP5823969B2 (ja
JP2013509168A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2010/066474 external-priority patent/WO2011051450A1/en
Publication of JP2013509168A publication Critical patent/JP2013509168A/ja
Publication of JP2013509168A5 publication Critical patent/JP2013509168A5/ja
Application granted granted Critical
Publication of JP5823969B2 publication Critical patent/JP5823969B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2012535849A 2009-10-29 2010-10-29 心臓特異的核酸調節因子ならびにこの方法および使用 Expired - Fee Related JP5823969B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP09174519.0 2009-10-29
EP09174519 2009-10-29
PCT/EP2010/066474 WO2011051450A1 (en) 2009-10-29 2010-10-29 Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Publications (3)

Publication Number Publication Date
JP2013509168A JP2013509168A (ja) 2013-03-14
JP2013509168A5 true JP2013509168A5 (enExample) 2013-12-19
JP5823969B2 JP5823969B2 (ja) 2015-11-25

Family

ID=43414855

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012535849A Expired - Fee Related JP5823969B2 (ja) 2009-10-29 2010-10-29 心臓特異的核酸調節因子ならびにこの方法および使用

Country Status (6)

Country Link
US (1) US9353164B2 (enExample)
EP (1) EP2494058B1 (enExample)
JP (1) JP5823969B2 (enExample)
AU (1) AU2010311376B2 (enExample)
CA (1) CA2778945A1 (enExample)
WO (1) WO2011051450A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5823969B2 (ja) 2009-10-29 2015-11-25 フエー・イー・ベー・フエー・ゼツト・ウエー 心臓特異的核酸調節因子ならびにこの方法および使用
US8859517B2 (en) * 2011-08-08 2014-10-14 Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT)
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
US9732345B2 (en) * 2013-12-09 2017-08-15 Baylor College Of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
CA2936726C (en) 2014-01-21 2024-04-30 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
US10570183B2 (en) * 2016-04-19 2020-02-25 The Board Of Regents Of The University Of Texas System Use of a small native peptide activator of SERCA pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload
US11920149B2 (en) 2017-03-27 2024-03-05 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
EP3847261A1 (en) * 2018-09-05 2021-07-14 Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft A method for engineering synthetic cis-regulatory dna
EP3870237A1 (en) 2018-10-26 2021-09-01 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
TW202129002A (zh) * 2019-12-09 2021-08-01 英商Ｕｃｌ商業有限責任公司 用於myh7關聯之心肌病之基因療法組合物及治療
CN115151646A (zh) * 2019-12-24 2022-10-04 阿斯克肋匹奥生物制药公司 调节核酸序列
CN115298307A (zh) 2020-02-18 2022-11-04 布鲁塞尔自由大学 核酸调节元件的新组合及其方法和用途
WO2022136388A1 (en) 2020-12-21 2022-06-30 Vrije Universiteit Brussel Cardiomyocyte-derived nucleic acid regulatory elements and methods and use thereof
AU2022307365A1 (en) 2021-07-08 2024-02-01 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2797273B1 (fr) 1999-08-06 2004-01-02 Pasteur Institut Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique
CA2396348A1 (en) 2000-01-14 2001-07-19 Beth Israel Deaconess Medical Center Cardiac-cell specific enhancer elements and uses thereof
ATE373095T1 (de) * 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
US20050004058A1 (en) * 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
US7714100B2 (en) * 2004-01-27 2010-05-11 Compugen Ltd Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of cardiac disease
WO2006015209A2 (en) * 2004-07-29 2006-02-09 Stem Cell Innovations, Inc. Differentiation of stem cells
US20080081354A1 (en) * 2006-10-02 2008-04-03 Cardiac Pacemakers, Inc. Devices, vectors and methods for inducible ischemia cardioprotection
WO2008054713A2 (en) 2006-10-30 2008-05-08 Thomas Jefferson University Tissue specific gene therapy treatment
WO2008126083A2 (en) * 2007-04-11 2008-10-23 Technion Research & Development Foundation Ltd. Methods for identification and selection of human embryonic stem cell derived cells
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
JP5823969B2 (ja) 2009-10-29 2015-11-25 フエー・イー・ベー・フエー・ゼツト・ウエー 心臓特異的核酸調節因子ならびにこの方法および使用

Similar Documents

Publication Publication Date Title
JP2013509168A5 (enExample)
JP2015521465A5 (enExample)
US9353164B2 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
JP2021505133A5 (enExample)
FI3313991T3 (fi) Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin
JP2020518268A5 (enExample)
ITTO20130669A1 (it) Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
JP2020054367A (ja) 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用
IL316189A (en) Treatment of complement-mediated disorders
JP2013518599A5 (enExample)
WO2014036524A4 (en) Genetically modified msc and therapeutic methods
JP2023544264A (ja) 核酸構築物、ウイルスベクター及びウイルス粒子
TW202421787A (zh) 保護心臟之心臟病療法
US20240350666A1 (en) Compositions and methods for high efficiency genome editing
WO2023147558A2 (en) Crispr methods for correcting bag3 gene mutations in vivo
JP6473438B2 (ja) プロモーター組成物
JP2016512028A5 (enExample)
Lai et al. Design of muscle gene therapy expression cassette
AU2014240247B2 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
WO2024215653A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
Ryu et al. Activation of the LMO2 gene in human lymphoid cells by targeted insertion of a single retroviral LTR expression cassette into the first intron
NZ761706A (en) Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
RU2013148606A (ru) Универсальный промотор для экспрессии терапевтических генов в клетках млекопитающих
NZ761706B2 (en) Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
HK1220486B (en) Promoter compositions