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Human Gene Therapy|February 14, 2022
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea PigJinghan Wang, Liping Zhao, Xi Gu, et al.
Human Gene Therapy|June 11, 2019
A New Era for Rare Genetic Diseases: Messenger RNA TherapyPaolo G V Martini, Lin T Guey
Human Gene Therapy|June 12, 2019
An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher MiceXiufang Pan, Scott A Sands, Yongping Yue, et al.
Human Gene Therapy|October 15, 2019
Silencing of GAS5 Alleviates Glaucoma in Rat Models by Reducing Retinal Ganglion Cell ApoptosisRong-Rong Zhou, Hai-Bo Li, Qi-Sheng You, et al.
Human Gene Therapy|May 16, 2019
Treatment of Cystathionine β-Synthase Deficiency in Mice Using a Minicircle-Based Naked DNA VectorHyung-Ok Lee, Lorena Gallego-Villar, Hiu Man Grisch-Chan, et al.
Human Gene Therapy|May 18, 2019
In Vitro Validation of a CRISPR-Mediated CFTR Correction Strategy for Preclinical Translation in PigsZhichang Peter Zhou, Liang Leo Yang, Huibi Cao, et al.
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