JP7500429B2 - 優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法 - Google Patents

優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法 Download PDF

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JP7500429B2
JP7500429B2 JP2020552905A JP2020552905A JP7500429B2 JP 7500429 B2 JP7500429 B2 JP 7500429B2 JP 2020552905 A JP2020552905 A JP 2020552905A JP 2020552905 A JP2020552905 A JP 2020552905A JP 7500429 B2 JP7500429 B2 JP 7500429B2
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ジー プリオリ シルビア
マッザンティ アンドレア
デネグリ マルコ
ナポリターノ カルロ
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イスティトゥティ クリニシ サイエンティフィシ マウジェリ エスピーエー エスビー
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    • A—HUMAN NECESSITIES
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JP2020552905A 2018-04-05 2019-04-05 優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法 Active JP7500429B2 (ja)

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IT102018000004253 2018-04-05
IT102018000004253A IT201800004253A1 (it) 2018-04-05 2018-04-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
PCT/IB2019/052817 WO2019193563A1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia

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MX2023000453A (es) * 2020-07-08 2023-02-09 Childrens Medical Center Polipeptidos de mybpc3 y usos de los mismos.
CN113957075B (zh) * 2021-11-22 2023-04-07 百世诺(北京)医学检验实验室有限公司 突变的遗传性心律失常基因及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017141157A1 (en) 2016-02-15 2017-08-24 Istituti Clinici Scientifici Maugeri Spa Sb Method of allele specific silencing for the treatment of autosomal dominant catecholaminergic polymorphic ventricular tachycardia (cpvt)
US20170360957A1 (en) 2011-08-08 2017-12-21 Istituti Clinici Scientifici Maugeri Spa Sb Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (cpvt)

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5766960A (en) 1987-07-27 1998-06-16 Australian Membrane And Biotechnology Research Institute Receptor membranes
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
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
MD1367C2 (ro) 1992-11-13 2000-11-30 Idec Pharmaceuticals Corporation Metode de tratament al limfomului celulelor B, anticorpi anti-CD20, hibridom.
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6268210B1 (en) 1998-05-27 2001-07-31 Hyseq, Inc. Sandwich arrays of biological compounds
US6232068B1 (en) 1999-01-22 2001-05-15 Rosetta Inpharmatics, Inc. Monitoring of gene expression by detecting hybridization to nucleic acid arrays using anti-heteronucleic acid antibodies
CA2407651C (en) 2000-04-27 2013-07-02 Max-Delbruck-Centrum Fur Molekulare Medizin Sleeping beauty, a transposon vector with a broad host range for the genetic transformation in vertebrates
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
US7741529B1 (en) 2006-05-04 2010-06-22 Priori Silvia G Transgenic animal model for catecholaminergic polymorphic ventricular tachycardia (CPVT) and use thereof
EP1995316A1 (de) 2007-05-25 2008-11-26 Qiagen GmbH Verfahren zur schonenden Zellaufreinigung, Zellgewinnung und Transfektion von Zellen
EP2009095A1 (en) 2007-06-28 2008-12-31 Innovalor AG Method of generating glucose-responsive cells
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
WO2010085699A2 (en) 2009-01-23 2010-07-29 The Johns Hopkins University Mammalian piggybac transposon and methods of use
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2016083625A1 (en) * 2014-11-28 2016-06-02 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nucleic acid encoding triadin for the prevention and/or treatment of heart failure

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170360957A1 (en) 2011-08-08 2017-12-21 Istituti Clinici Scientifici Maugeri Spa Sb Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (cpvt)
WO2017141157A1 (en) 2016-02-15 2017-08-24 Istituti Clinici Scientifici Maugeri Spa Sb Method of allele specific silencing for the treatment of autosomal dominant catecholaminergic polymorphic ventricular tachycardia (cpvt)

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Circulation，2014年，129，pp.2673-2681
Heart Rhythm，2016年，13，pp.1652-1660

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IL315337A (en) 2024-10-01
JP2024119900A (ja) 2024-09-03
IL277635A (en) 2020-11-30
AU2019249374A1 (en) 2020-10-22
JP2021523093A (ja) 2021-09-02
IL277635B2 (en) 2025-02-01
DK3773744T3 (da) 2024-08-26
EP4356727A3 (en) 2024-07-24
FI3773744T3 (fi) 2024-08-12
ES2988961T3 (es) 2024-11-22
AU2026202418A1 (en) 2026-04-16
EP3773744A1 (en) 2021-02-17
US20210030894A1 (en) 2021-02-04
AU2019249374B2 (en) 2026-01-08
WO2019193563A1 (en) 2019-10-10
PL3773744T3 (pl) 2024-11-04
EP3773744B1 (en) 2024-05-22
EP4356727A2 (en) 2024-04-24
CA3094141A1 (en) 2019-10-10
PT3773744T (pt) 2024-08-22
IT201800004253A1 (it) 2019-10-05
IL277635B1 (en) 2024-10-01

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