Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration

Preclinical studies have demonstrated that a single injection of an adeno-associated virus (AAV) vector into the cerebrospinal fluid (CSF) can achieve widespread gene transfer throughout the central nervous system. Successfully translating this approach to humans requires identifying factors that in...

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Bibliographic Details
Main Authors: Christian Hinderer, Nathan Katz, Cecilia Dyer, Tamara Goode, Julia Johansson, Peter Bell, Laura Richman, Elizabeth Buza, James M. Wilson
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300723