HK1254639A1 - Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions - Google Patents

Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions Download PDF

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Publication number
HK1254639A1
HK1254639A1 HK18113606.9A HK18113606A HK1254639A1 HK 1254639 A1 HK1254639 A1 HK 1254639A1 HK 18113606 A HK18113606 A HK 18113606A HK 1254639 A1 HK1254639 A1 HK 1254639A1
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Hong Kong
Prior art keywords
vector
cell
expression
paracrine
igfi
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HK18113606.9A
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English (en)
Chinese (zh)
Inventor
Kirk H. HAMMOND
Hua Mei GAO
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The Regents Of The University Of California
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Publication of HK1254639A1 publication Critical patent/HK1254639A1/en

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    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
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HK18113606.9A 2012-02-14 2018-10-24 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions HK1254639A1 (en)

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WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
BR112014020127A2 (pt) * 2012-02-14 2020-06-30 The Regents Of The University Of Calefornia métodos para tratamento, melhoramento ou proteção (prevenção) de um indivíduo ou um paciente, e de uma disfunção, uma doença, uma infecção ou uma condição, e, método para suprimir o ganho de peso, ou suprimir o apepite, ou estimular ou iniciar a perda de peso
AU2015242354A1 (en) * 2014-04-03 2016-11-10 The Regents Of The University Of California Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunction and congestive heart failure
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CA2964272A1 (en) * 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
RU2716422C2 (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
EP3294752B1 (en) * 2015-05-12 2020-11-04 The Regents of the University of California Peptide treatment for inflammation and fibrosis
CN113999875A (zh) * 2015-05-28 2022-02-01 康奈尔大学 腺相关病毒介导的c1ei递送作为用于血管性水肿的疗法
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CA3252099A1 (en) 2016-05-18 2025-06-05 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018071831A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
BR112019015081A2 (pt) * 2017-01-23 2020-03-10 Rutgers, The State University Of New Jersey Composições para reduzir a expressão da sarcolipina e prevenir e tratar a distrofia muscular e a cardiomiopatia e os métodos de utilização
CN119236118A (zh) * 2017-02-28 2025-01-03 基因药物株式会社 含肿瘤特异性溶瘤腺病毒和免疫检查点抑制剂的抗癌组合物
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
US11760987B2 (en) 2017-09-07 2023-09-19 The Regents Of The University Of California Compositions and methods for the treatment or prevention of heart failure
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
CN119242711A (zh) 2017-10-16 2025-01-03 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
KR102785135B1 (ko) * 2018-06-11 2025-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 눈 질환을 치료하는 탈메틸화
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN115843313B (zh) * 2020-06-12 2025-09-05 吉赛尔美德公司 用于多重靶向的重组单纯疱疹病毒及其用途
CN112826823B (zh) * 2021-01-11 2022-03-08 中国人民解放军军事科学院军事医学研究院 多西环素在制备治疗和/或预防和/或缓解和/或改善心肌病药物中的应用

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5217879A (en) 1989-01-12 1993-06-08 Washington University Infectious Sindbis virus vectors
US5733542A (en) 1990-11-16 1998-03-31 Haynesworth; Stephen E. Enhancing bone marrow engraftment using MSCS
US5270192A (en) 1991-02-07 1993-12-14 Monsanto Company Biological artificial liver
US5846743A (en) 1995-02-22 1998-12-08 Brigham And Women's Hospital, Inc. Polyphoshoinositide binding peptides for intracellular drug delivery
US20020103147A1 (en) * 1997-09-05 2002-08-01 Hammond H. Kirk Gene therapy for congestive heart failure
US7361332B2 (en) 1995-03-17 2008-04-22 The Regents Of The University Of California Treating tumors using implants comprising combinations of allogeneic cells
US6214797B1 (en) * 1995-06-13 2001-04-10 The Salk Institute For Biological Studies Urocortin peptides, nucleic acid encoding same methods for using same
WO1997016533A1 (en) 1995-10-31 1997-05-09 The Regents Of The University Of California Mammalian artificial chromosomes and methods of using same
PT876165E (pt) 1995-12-18 2006-10-31 Angiotech Biomaterials Corp Composicoes de polimeros reticulados e processos para a sua utilizacao
US6025155A (en) 1996-04-10 2000-02-15 Chromos Molecular Systems, Inc. Artificial chromosomes, uses thereof and methods for preparing artificial chromosomes
US6933331B2 (en) 1998-05-22 2005-08-23 Nanoproducts Corporation Nanotechnology for drug delivery, contrast agents and biomedical implants
US5874268A (en) 1996-09-23 1999-02-23 Duke University Method of introducing exogenous compounds into cells by electroporation and apparatus for same
WO1998020027A2 (en) * 1996-11-01 1998-05-14 Eurogene Limited Therapeutic use of an agent that stimulates no or prostacyclin production and delivery device
US6630137B1 (en) 1997-04-28 2003-10-07 Eli Lilly And Company Activated protein C formulations
CA2289600C (en) * 1997-05-06 2010-06-29 The Regents Of The University Of California Techniques and compositions for treating heart failure and ventricular remodeling by in vivo delivery of angiogenic transgenes
US20050095227A1 (en) * 1997-07-22 2005-05-05 The General Hospital Corporation Treating heart failure
SE9704076D0 (sv) 1997-11-06 1997-11-06 Holdingbolaget Vid Goeteborgs Method for permeabilisation of cell structures and use thereof
US6886568B2 (en) 1998-04-08 2005-05-03 The Johns Hopkins University Method for fabricating cell-containing implants
ES2313784T3 (es) * 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US6589503B1 (en) 1998-06-20 2003-07-08 Washington University Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy
US6958212B1 (en) 1999-02-01 2005-10-25 Eidgenossische Technische Hochschule Zurich Conjugate addition reactions for the controlled delivery of pharmaceutically active compounds
CN1433325A (zh) * 1999-11-05 2003-07-30 加利福尼亚大学董事会 通过体内基因递送治疗心血管疾病的技术和组合物
US6759386B2 (en) 2000-04-06 2004-07-06 Wayne P. Franco Methods of use of fibroblast growth factor, vascular endothelial growth factor and related proteins in the treatment of acute and chronic heart disease
US7442390B2 (en) 2000-06-05 2008-10-28 University Of South Florida Method for enhancing engraftment of cells using mesenchymal progenitor cells
NZ523803A (en) * 2000-08-04 2004-08-27 Res Dev Foundation Human urocortin-related polypeptide (URP) and uses thereof to rreat a pathophysiological state, eg stress, anxiety and appetite dysfunction
ES2313978T3 (es) * 2000-08-04 2009-03-16 Research Development Foundation Proteinas urocortina y sus usos.
EP1559777A1 (en) 2001-04-24 2005-08-03 Hokkaido Technology Licensing Office Co., Ltd. Process for maturating small heptocyte-rich colonies into liver tissue
PT2359869T (pt) * 2001-12-17 2019-04-16 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US6936585B2 (en) * 2002-01-16 2005-08-30 The Procter & Gamble Company Corticotropin releasing factor 2 receptor agonists
US7379765B2 (en) 2003-07-25 2008-05-27 Dexcom, Inc. Oxygen enhancing membrane systems for implantable devices
US7166133B2 (en) 2002-06-13 2007-01-23 Kensey Nash Corporation Devices and methods for treating defects in the tissue of a living being
US20040151766A1 (en) 2003-01-30 2004-08-05 Monahan Sean D. Protein and peptide delivery to mammalian cells in vitro
US7241455B2 (en) 2003-04-08 2007-07-10 Boston Scientific Scimed, Inc. Implantable or insertable medical devices containing radiation-crosslinked polymer for controlled delivery of a therapeutic agent
US7794706B2 (en) 2003-10-14 2010-09-14 Medivas, Llc Bioactive wound dressings and implantable devices and methods of use
US20050136121A1 (en) 2003-12-22 2005-06-23 Shear/Kershman Laboratories, Inc. Oral peptide delivery system with improved bioavailability
CA2549966A1 (en) 2003-12-24 2005-07-07 Ltt Bio-Pharma Co., Ltd. Drug-containing nanoparticle, process for producing the same and parenterally administered preparation from the nanoparticle
US20050244463A1 (en) 2004-04-30 2005-11-03 Allergan, Inc. Sustained release intraocular implants and methods for treating ocular vasculopathies
US8658203B2 (en) 2004-05-03 2014-02-25 Merrimack Pharmaceuticals, Inc. Liposomes useful for drug delivery to the brain
EP1773298A1 (en) 2004-08-06 2007-04-18 Biospectrum, Inc. Multiple layered liposome and preparation method thereof
WO2006023803A2 (en) 2004-08-20 2006-03-02 Artes Medical, Inc. Methods of administering microparticles combined with autologous body components
US7919112B2 (en) 2004-08-26 2011-04-05 Pathak Holdings, Llc Implantable tissue compositions and method
US7351423B2 (en) 2004-09-01 2008-04-01 Depuy Spine, Inc. Musculo-skeletal implant having a bioactive gradient
US7501486B2 (en) 2004-09-07 2009-03-10 Burnham Institute For Medical Research Peptides that selectively home to heart vasculature and related conjugates and methods
EP1812074B1 (en) 2004-10-18 2010-12-29 Nitto Denko Corporation Intracellular peptide delivery
WO2006086402A2 (en) * 2005-02-08 2006-08-17 Research Development Foundation Compositions and methods related to soluble g-protein coupled receptors(sgpcrs)
US7759120B2 (en) 2005-03-02 2010-07-20 Kps Bay Medical, Inc. Seeding implantable medical devices with cells
EP1865992A2 (en) * 2005-03-31 2007-12-19 Mytogen Inc. Treatment for heart disease
AU2006274413B2 (en) 2005-07-27 2013-01-10 Arbutus Biopharma Corporation Systems and methods for manufacturing liposomes
US20070265221A1 (en) * 2006-05-09 2007-11-15 Weiss Robert G Methods to improve creatine kinase metabolism and contractile function in cardiac muscle for the treatment of heart failure
WO2008047241A2 (en) * 2006-10-16 2008-04-24 Conjuchem Biotechnologies Inc. Modified corticotropin releasing factor peptides and uses thereof
AU2007200990B8 (en) 2006-11-21 2011-05-12 Living Cell Technologies Limited Cell Implantation To Prevent and/or Treat Hearing Loss
JP2011503207A (ja) * 2007-11-16 2011-01-27 サン ディエゴ ステート ユニバーシティ リサーチ ファウンデーション 循環系細胞におけるpim−1活性を操作するための組成物および方法
NZ587060A (en) * 2007-12-31 2012-09-28 Nanocor Therapeutics Inc Rna interference for the treatment of heart failure
MX336145B (es) * 2008-05-16 2016-01-08 Corthera Inc Metodo para el tratamiento de insuficiencia cardiaca cronica.
EA022948B1 (ru) * 2008-05-16 2016-03-31 Кортера, Инк. Лечение релаксином одышки, ассоциированной с острой сердечной недостаточностью
US10040838B2 (en) * 2008-11-04 2018-08-07 Janssen Pharmaceutica Nv CRHR2 peptide agonists and uses thereof
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
PH12012500895A1 (en) * 2009-11-04 2012-11-12 Janssen Pharmaceutica Nv Method for treating heart failure with stresscopin-like peptides
BR112014020127A2 (pt) * 2012-02-14 2020-06-30 The Regents Of The University Of Calefornia métodos para tratamento, melhoramento ou proteção (prevenção) de um indivíduo ou um paciente, e de uma disfunção, uma doença, uma infecção ou uma condição, e, método para suprimir o ganho de peso, ou suprimir o apepite, ou estimular ou iniciar a perda de peso
ES2926977T3 (es) * 2016-01-19 2022-10-31 Univ California Métodos para el tratamiento de la enfermedad de Danon y otros trastornos de autofagia

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