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Nature Communications|May 23, 2026
Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in miceXiupeng Chen, Qing Xie, Sarah J Nath, et al.Human Gene Therapy|June 15, 2016
Naturally Existing Oncolytic Virus M1 Is Nonpathogenic for the Nonhuman Primates After Multiple Rounds of Repeated Intravenous InjectionsHaipeng Zhang, Yuan Lin, Kai Li, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|January 26, 2025
A comprehensive atlas of AAV tropism in the mouseChristopher J Walkey, Kathy J Snow, Jote Bulcha, et al.Biorxiv : the Preprint Server for Biology|September 24, 2024
A Comprehensive Atlas of AAV Tropism in the MouseChristopher J Walkey, Kathy J Snow, Jote Bulcha, et al.Human Gene Therapy|May 3, 2022
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and ObstaclesElisabeth M W Eekhoff, Ruben D de Ruiter, Bernard J Smilde, et al.Nature Biotechnology|November 24, 2022
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrasesMatthew T N Yarnall, Eleonora I Ioannidi, Cian Schmitt-Ulms, et al.Science Translational Medicine|February 26, 2025
BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine diseaseJiaming Wang, Laura E Poskitt, Jillian Gallagher, et al.Human Gene Therapy|July 25, 2024
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus ArticleJan C Klapwijk, Alberto Del Rio Espinola, Silvana Libertini, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 7, 2025
Current clinical applications of AAV-mediated gene therapyBarry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.Human Gene Therapy|October 3, 2014
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard materialEduard Ayuso, Véronique Blouin, Martin Lock, et al.Pageof 33