Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration-Reference-Cited by-同舟云学术

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Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration

Published:2020-06 Issue: Volume:17 Page:969-974
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Hinderer Christian,Katz Nathan,Dyer Cecilia,Goode Tamara,Johansson Julia,Bell Peter,Richman Laura,Buza Elizabeth,Wilson James M.

Funder

Biogen

University of Pennsylvania

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference19 articles.

1. Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson’s disease;Mittermeyer;Hum. Gene Ther.,2012

2. Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial;Tardieu;Lancet Neurol.,2017

3. Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency;Kojima;Brain,2019

4. Effective gene therapy for an inherited CNS disease in a large animal model;Vite;Ann. Neurol.,2005

5. Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN;Hinderer;Hum. Gene Ther.,2018

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