Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products

Affiliations

¹ Department of Pediatrics and Molecular Genetics & Microbiology, College of Medicine, University of Florida, Gainesville 32611, FL, USA. Electronic address: bbyrne@ufl.edu.

Comment

Barry J Byrne. Mol Ther. 2018.

. 2018 Mar 7;26(3):669-671.

doi: 10.1016/j.ymthe.2018.02.009. Epub 2018 Mar 1.

¹ Department of Pediatrics and Molecular Genetics & Microbiology, College of Medicine, University of Florida, Gainesville 32611, FL, USA. Electronic address: bbyrne@ufl.edu.

No abstract available

Comment on

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.
Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, Richman LK, Wilson JM. Hinderer C, et al. Hum Gene Ther. 2018 Mar;29(3):285-298. doi: 10.1089/hum.2018.015. Epub 2018 Feb 12. Hum Gene Ther. 2018. PMID: 29378426 Free PMC article.
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.
Hordeaux J, Wang Q, Katz N, Buza EL, Bell P, Wilson JM. Hordeaux J, et al. Mol Ther. 2018 Mar 7;26(3):664-668. doi: 10.1016/j.ymthe.2018.01.018. Epub 2018 Feb 2. Mol Ther. 2018. PMID: 29428298 Free PMC article.

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