Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8
Inagaki, Katsuya, Fuess, Sally, Storm, Theresa A., Gibson, Gregory A., Mctiernan, Charles F., Kay, Mark A., Nakai, Hiroyuki·2006·Molecular Therapy
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- A Translational Pathway for Recombinant Adeno-Associated Virus Human Gene Therapy: From Target Identification and Animal Modeling of the Disease to Non-Human Primate and Human StudiesAlisha M. Gruntman · 2016 · The Journal of the American Medical Association (JAMA) Network (American Medical Association)
- Gene therapy in mouse models of spinal muscular atrophy :||using SMN gene replacement to address biological questionsJacqueline Glascock · 2013
- UNCOVERING STRUCTURAL COMPONENTS OF THE ADENO-ASSOCIATED VIRAL CAPSID THAT CAN BE MODIFIED TO IMPROVE CLINICAL GENE THERAPY OUTCOMESJayme K. Warischalk · 2019 · Carolina Digital Repository (University of North Carolina at Chapel Hill)
- Experimental studies on retinoic acid signaling in myocardial remodelling and remote gene therapy using heme oxygenase-1Dusan Bilbija · 2012
- 基于CRISPR/Cas9基因编辑技术的人类遗传疾病基因治疗相关研究进展可 门, 醒妹 段, 阳 杨, 于全 魏 · 2017 · Scientia Sinica Vitae
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- Paracrine Engineering of Human Cardiac Stem Cells with Insulin-Like Growth Factor 1 Promotes Cell Survival to Enhance Myocardial RepairRobyn Jackson · 2014 · uO Research (University of Ottawa)
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- CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular DystrophySimon Guiraud, Sumitava Dastidar, Fetta Mazed, Fatima Amor, Maëlle Ralu, Anne De Cian, Isabelle Richard, Giuseppe Ronzitti, Francesco Saverio Tedesco, Mario Amendola · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
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