HK1203845A1 - 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

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Publication number
HK1203845A1
HK1203845A1 HK15104654.2A HK15104654A HK1203845A1 HK 1203845 A1 HK1203845 A1 HK 1203845A1 HK 15104654 A HK15104654 A HK 15104654A HK 1203845 A1 HK1203845 A1 HK 1203845A1
Authority
HK
Hong Kong
Prior art keywords
paracrine
genes
conditions
cardiovascular diseases
systemic delivery
Prior art date
Application number
HK15104654.2A
Other languages
English (en)
Chinese (zh)
Inventor
H Kirk Hammond
Mei Hua Gao
Original Assignee
Univ California
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ California filed Critical Univ California
Publication of HK1203845A1 publication Critical patent/HK1203845A1/xx

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A61K31/4353—Heterocyclic 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
    • A61K31/436—Heterocyclic 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 the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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HK15104654.2A 2012-02-14 2015-05-15 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions HK1203845A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261598772P 2012-02-14 2012-02-14
PCT/US2013/025997 WO2013123094A2 (en) 2012-02-14 2013-02-13 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions

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HK1203845A1 true HK1203845A1 (en) 2015-11-06

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Application Number Title Priority Date Filing Date
HK15104654.2A HK1203845A1 (en) 2012-02-14 2015-05-15 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
HK18113606.9A HK1254639A1 (zh) 2012-02-14 2018-10-24 用於心血管疾病和其他病況的旁分泌基因的全身性遞送和受調節的表達

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HK18113606.9A HK1254639A1 (zh) 2012-02-14 2018-10-24 用於心血管疾病和其他病況的旁分泌基因的全身性遞送和受調節的表達

Country Status (18)

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US (3) US20150118287A1 (xx)
EP (2) EP3311830A1 (xx)
JP (3) JP6562632B2 (xx)
KR (1) KR102107482B1 (xx)
CN (2) CN108310396B (xx)
AU (2) AU2013221615B2 (xx)
BR (1) BR112014020127A2 (xx)
CA (1) CA2864100A1 (xx)
DK (1) DK2814513T3 (xx)
ES (1) ES2660492T3 (xx)
HK (2) HK1203845A1 (xx)
HU (1) HUE036640T2 (xx)
IL (3) IL234007A0 (xx)
IN (1) IN2014DN06789A (xx)
PL (1) PL2814513T3 (xx)
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Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP3311830A1 (en) * 2012-02-14 2018-04-25 The Regents of The University of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
CA2943751A1 (en) * 2014-04-03 2015-10-08 The Regents Of The University Of California Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunctions and congestive heart failure
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
JP7023108B2 (ja) 2014-10-21 2022-02-21 ユニバーシティ オブ マサチューセッツ 組み換えａａｖバリアントおよびその使用
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
JP2017535266A (ja) 2014-11-14 2017-11-30 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）を治療する組成物および方法
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
AU2016260190A1 (en) * 2015-05-12 2017-11-23 The Regents Of The University Of California Peptide treatment for inflammation and fibrosis
CN108025047B (zh) * 2015-05-28 2021-11-19 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
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
MX2019008679A (es) * 2017-01-23 2019-11-18 Univ Rutgers Composiciones para reducir la expresion de sarcolipina y prevenir y tratar la distrofia muscular y la miocardiopatia y metodos de uso.
EP3591041A4 (en) * 2017-02-28 2021-01-06 Genemedicine Co., Ltd. ANTI-CANCER COMPOSITION CONSISTING OF A TUMOR-SPECIFIC ONCOLYTIC ADENOVIRUS AND AN IMMUNE CHECKPOINT INHIBITOR
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
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
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7089603B2 (ja) * 2018-06-11 2022-06-22 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 眼疾患を治療するための脱メチル化
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
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
US5721118A (en) 1995-10-31 1998-02-24 The Regents Of The University Of California, San Diego Mammalian artificial chromosomes and methods of using same
EP2111876B1 (en) 1995-12-18 2011-09-07 AngioDevice International GmbH Crosslinked polymer compositions and methods for their use
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
EP0941116B1 (en) * 1996-11-01 2005-01-19 Ark Therapeutics Limited Use of vegf for the manufacture of a medicament for the treatment or prevention of intimal hyperplasia and delivery device
US6630137B1 (en) 1997-04-28 2003-10-07 Eli Lilly And Company Activated protein C formulations
KR20070005030A (ko) * 1997-05-06 2007-01-09 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 혈관형성성 트랜스유전자의 생체내 송달에 의한 심장 질환및 심실 리모델링을 치료하기 위한 방법 및 조성물
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
JP4060531B2 (ja) * 1998-05-28 2008-03-12 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
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
NZ546670A (en) * 1999-11-05 2009-02-28 Univ California Techniques and compositions for treating cardiovascular disease by in vivo gene delivery
WO2001048164A2 (en) * 1999-12-27 2001-07-05 The Regents Of The University Of California Modified adenylylcyclase type vi useful in gene therapy for congestive heart failure
WO2001077328A1 (en) 2000-04-06 2001-10-18 Franco Wayne P Methods of using growth factors for treating heart disease
US7442390B2 (en) 2000-06-05 2008-10-28 University Of South Florida Method for enhancing engraftment of cells using mesenchymal progenitor cells
AU8468301A (en) * 2000-08-04 2002-02-18 Res Dev Foundation Urocortin proteins and uses thereof
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
JPWO2002088332A1 (ja) 2001-04-24 2004-08-19 北海道ティー・エル・オー株式会社 小型肝細胞高含有コロニー、その調製方法、その肝組織への成熟化方法、成熟化した小型肝細胞高含有コロニーを用いた薬物機能の推定方法
AU2002360291A1 (en) * 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
US7192923B2 (en) * 2002-01-16 2007-03-20 The Procter & Gamble Company Corticotropin releasing factor 2 receptor agonists
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
JP4708342B2 (ja) 2003-07-25 2011-06-22 デックスコム・インコーポレーテッド 埋設可能な装置に用いる酸素増大膜システム
US7794706B2 (en) 2003-10-14 2010-09-14 Medivas, Llc Bioactive wound dressings and implantable devices and methods of use
WO2005063156A1 (en) 2003-12-22 2005-07-14 Shear/Kershman Laboratories, Inc. Oral peptide delivery system with improved bioavailability
JP3903061B2 (ja) 2003-12-24 2007-04-11 株式会社Ｌｔｔバイオファーマ 薬物を含有するナノ粒子およびその製造方法、ならびに当該ナノ粒子からなる非経口投与用製剤
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
WO2006014035A1 (en) 2004-08-06 2006-02-09 Biospectrum, Inc. Multiple layered liposome and preparation method thereof
CA2569736A1 (en) 2004-08-20 2006-03-02 Russell Anderson 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
DE602005025676D1 (de) 2004-10-18 2011-02-10 Nitto Denko Corp Intrazelluläre peptidabgabe
KR20070112155A (ko) * 2005-02-08 2007-11-22 리서치 디벨럽먼트 파운데이션 가용성 Ｇ-단백질 연결 수용체(ｓＧＰＣＲ)와 관련된조성물 및 방법
US7759120B2 (en) 2005-03-02 2010-07-20 Kps Bay Medical, Inc. Seeding implantable medical devices with cells
WO2006121532A2 (en) * 2005-03-31 2006-11-16 Mytogen, Inc. Treatment for heart disease
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
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
US7982018B2 (en) * 2006-10-16 2011-07-19 Conjuchem, Llc Modified corticotropin releasing factor peptides and uses thereof
US20080119909A1 (en) 2006-11-21 2008-05-22 Neurotrophincell Pty Limited Cell implantation to prevent and/or treat hearing loss
CA2705862C (en) * 2007-11-16 2018-03-27 San Diego State University Research Foundation Compositions and method for manipulating pim-1 activity in circulatory system cells
CN101970051A (zh) 2007-12-31 2011-02-09 纳诺科尔治疗公司 用于治疗心力衰竭的rna干扰
WO2009140657A2 (en) * 2008-05-16 2009-11-19 Corthera, Inc. Method of treating chronic heart failure
CA2724540C (en) * 2008-05-16 2014-07-08 Corthera, Inc. Treating dyspnea associated with acute heart failure with relaxin
EA201170647A1 (ru) * 2008-11-04 2011-12-30 Янссен Фармацевтика Нв Пептиды-агонисты crhr2 и их использование
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CN102711801A (zh) * 2009-11-04 2012-10-03 詹森药业有限公司 用顶压素样肽治疗心力衰竭的方法
EP3311830A1 (en) * 2012-02-14 2018-04-25 The Regents of The University of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
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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