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