Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality-Reference-Cited by-同舟云学术

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Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

Published:2019-03 Issue:5 Volume:101 Page:839-862
ISSN:0896-6273
Container-title:Neuron
language:en
Short-container-title:Neuron

Author:

Hudry Eloise,Vandenberghe Luk H.

Funder

Foundation Fighting Blindness

Research to Prevent Blindness

Lonza Houston

Oxford Biomedica

Butler Family Foundation

Candyce Henwood Fund

Ush2a Consortium

National Institutes of Health

Publisher

Elsevier BV

Subject

General Neuroscience

Reference241 articles.

1. Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy;Akil;Neuron,2012

2. Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness;Amado;Sci. Transl. Med.,2010

3. Increasing N-acetylaspartate in the brain during postnatal myelination does not cause the CNS pathologies of Canavan disease;Appu;Front. Mol. Neurosci.,2017

4. Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy;Armbruster;Mol. Ther. Methods Clin. Dev.,2016

5. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model;Auricchio;Hum. Mol. Genet.,2001

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1. AAV-aMTD-Parkin, a therapeutic gene delivery cargo, enhances motor and cognitive functions in Parkinson's and Alzheimer’s diseases;Pharmacological Research;2024-10

2. Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations;Molecular Therapy - Nucleic Acids;2024-09

3. Emerging Gene Therapies for Alzheimer’s and Parkinson’s Diseases: An Overview of Clinical Trials and Promising Candidates;Cureus;2024-08-16

4. Current developments of gene therapy in human diseases;MedComm;2024-08-16

5. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12

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