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Human Gene Therapy|May 11, 2023
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's AtaxiaCarlos Munoz-Zuluaga, Monica Gertz, Melissa Yost-Bido, et al.
Human Gene Therapy|September 25, 2012
Enhanced function of redirected human T cells expressing linker for activation of T cells that is resistant to ubiquitylationNaoki Kunii, Yangbing Zhao, Shuguang Jiang, et al.
Human Gene Therapy|July 29, 2020
Gene Editing for Treatment and Prevention of Human Diseases: A Global Survey of Gene Editing-Related ResearchersLeonardo Fernandes Moutinho Rocha, Luiza Amara Maciel Braga, Fabio Batista Mota
Human Gene Therapy|March 27, 2015
Improved in vitro human tumor models for cancer gene therapyPhilip G Jakeman, Thomas E Hills, Alison B Tedcastle, et al.
Human Gene Therapy|May 16, 2014
Development of therapeutic splice-switching oligonucleotidesPetra Disterer, Adrianna Kryczka, Yuqi Liu, et al.
Human Gene Therapy|May 22, 2014
Gene therapy: a promising approach to treating spinal muscular atrophyPádraig J Mulcahy, Kayleigh Iremonger, Evangelia Karyka, et al.
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