Commentary: Can the new synthetic adeno-associated virus vector deliver the promise of cardiac gene therapy?

Journal of Thoracic and Cardiovascular Surgery ◽

10.1016/j.jtcvs.2021.06.022 ◽

2021 ◽

Author(s):

Ming Lau ◽

Michael K.Y. Hsin

Keyword(s):

Gene Therapy ◽

Virus Vector ◽

Adeno Associated Virus ◽

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69. Gene therapy of mucopolysaccharidosis type I: Intraventricular administration of adeno-associated virus vector transducing the human alpha-l-iduronidase gene in a murine model of the disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2007.10.081 ◽

2008 ◽

Vol 93 (2) ◽

pp. 31

Author(s):

R. Scott McIvor ◽

Daniel A. Wolf ◽

Zhenhong Nan ◽

Mayra Quito ◽

Kelly Podetz-Pedersen ◽

...

Keyword(s):

Gene Therapy ◽

Murine Model ◽

Virus Vector ◽

Type I ◽

Intraventricular Administration ◽

Mucopolysaccharidosis Type ◽

Mucopolysaccharidosis Type I ◽

Adeno Associated Virus

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Reversal of gene expression profile in the phenylketonuria mouse model after adeno-associated virus vector-mediated gene therapy

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2005.06.015 ◽

2005 ◽

Vol 86 ◽

pp. 124-132 ◽

Author(s):

Hyun-Jeong Oh ◽

Hyejin Lee ◽

Joo-Won Park ◽

Hwanseok Rhee ◽

Soo Kyung Koo ◽

...

Keyword(s):

Gene Expression ◽

Gene Therapy ◽

Mouse Model ◽

Gene Expression Profile ◽

Expression Profile ◽

Virus Vector ◽

Adeno Associated Virus

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Translational Aspects of Adeno-Associated Virus–Mediated Cardiac Gene Therapy

Human Gene Therapy ◽

10.1089/hum.2017.229 ◽

2018 ◽

Vol 29 (12) ◽

pp. 1341-1351 ◽

Author(s):

Tilman Ziegler ◽

Kiyotake Ishikawa ◽

Rabea Hinkel ◽

Christian Kupatt

Keyword(s):

Gene Therapy ◽

Adeno Associated Virus ◽

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Exogenous control of cardiac gene therapy: Evidence of regulated myocardial transgene expression after adenovirus and adeno-associated virus transfer of expression cassettes containing corticosteroid response element promoters

Journal of Thoracic and Cardiovascular Surgery ◽

10.1016/s0022-5223(99)70137-6 ◽

1999 ◽

Vol 118 (1) ◽

pp. 26-34 ◽

Author(s):

Leonard Y. Lee ◽

Xiaohuai Zhou ◽

Dean R. Polce ◽

Tarek El-Sawy ◽

Shailen R. Patel ◽

...

Keyword(s):

Gene Therapy ◽

Transgene Expression ◽

Response Element ◽

Adeno Associated Virus ◽

Virus Transfer ◽

Cardiac Gene ◽

Corticosteroid Response ◽

Exogenous Control

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Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

Neuromuscular Disorders ◽

10.1016/j.nmd.2015.06.458 ◽

2015 ◽

Vol 25 ◽

pp. S315 ◽

Author(s):

C. Le Guiner ◽

L. Servais ◽

M. Montus ◽

F. Bodvael ◽

B. Gjata ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Muscle Function ◽

Canine Model ◽

Virus Vector ◽

Adeno Associated Virus

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Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia

Gene Therapy ◽

10.1038/sj.gt.3302903 ◽

2007 ◽

Vol 14 (3) ◽

pp. 281-281

Author(s):

D D Koeberl ◽

B D Sun ◽

T V Damodaran ◽

T Brown ◽

D S Millington ◽

...

Keyword(s):

Gene Therapy ◽

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Virus Vector ◽

Adeno Associated Virus ◽

Sustained Efficacy ◽

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Cardiac gene therapy with adeno-associated virus-based vectors

Current Opinion in Cardiology ◽

10.1097/hco.0000000000000386 ◽

2017 ◽

Vol 32 (3) ◽

pp. 275-282 ◽

Author(s):

Kyle Chamberlain ◽

Jalish M. Riyad ◽

Thomas Weber

Keyword(s):

Gene Therapy ◽

Adeno Associated Virus ◽

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Adeno-associated virus vector-mediated bcl-2 gene transfer into post-ischemic gerbil brain in vivo: prospects for gene therapy of ischemia-induced neuronal death

Gene Therapy ◽

10.1038/sj.gt.3301211 ◽

2000 ◽

Vol 7 (14) ◽

pp. 1244-1249 ◽

Author(s):

K Shimazaki ◽

M Urabe ◽

J Monahan ◽

K Ozawa ◽

N Kawai

Keyword(s):

Gene Therapy ◽

Gene Transfer ◽

Neuronal Death ◽

Virus Vector ◽

Adeno Associated Virus ◽

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Feasibility of gene therapy in Gaucher disease using an adeno-associated virus vector

Journal of Human Genetics ◽

10.1007/s10038-004-0186-8 ◽

2004 ◽

Vol 49 (10) ◽

pp. 536-543 ◽

Author(s):

Young Bin Hong ◽

Eun Young Kim ◽

Han-Wook Yoo ◽

Sung-Chul Jung

Keyword(s):

Gene Therapy ◽

Gaucher Disease ◽

Virus Vector ◽

Adeno Associated Virus

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Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia

JAMA Ophthalmology ◽

10.1001/jamaophthalmol.2019.3278 ◽

2019 ◽

Vol 137 (11) ◽

pp. 1247 ◽

Author(s):

M. Dominik Fischer ◽

G. Alex Ochakovski ◽

Benjamin Beier ◽

Immanuel P. Seitz ◽

Yousof Vaheb ◽

...

Keyword(s):

Gene Therapy ◽

Virus Vector ◽

Efficacy And Safety ◽

Adeno Associated Virus ◽

Retinal Gene Therapy

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