Selection of an Efficient AAV Vector for Robust CNS Transgene Expression-Reference-Cited by-同舟云学术

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Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

Published:2019-12 Issue: Volume:15 Page:320-332
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Hanlon Killian S.,Meltzer Jonah C.,Buzhdygan Tetyana,Cheng Ming J.,Sena-Esteves Miguel,Bennett Rachel E.,Sullivan Timothy P.,Razmpour Roshanak,Gong Yi,Ng Carrie,Nammour Josette,Maiz Daniela,Dujardin Simon,Ramirez Servio H.,Hudry Eloise,Maguire Casey A.

Funder

NIH

NIDCD

NIA

Eunice Kennedy Shriver National Institute of Child Health & Human Development

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference26 articles.

1. Single-dose gene-replacement therapy for spinal muscular atrophy;Mendell;N. Engl. J. Med.,2017

2. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response;Manno;Nat. Med.,2006

3. 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

4. Bioengineered viral platform for intramuscular passive vaccine delivery to human skeletal muscle;Paulk;Mol. Ther. Methods Clin. Dev.,2018

5. Bioengineered AAV capsids with combined high human liver transduction in vivo and unique humoral seroreactivity;Paulk;Mol. Ther.,2018

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2. Systematic multi-trait AAV capsid engineering for efficient gene delivery;Nature Communications;2024-08-04

3. Therapeutic Application and Structural Features of Adeno-Associated Virus Vector;Current Issues in Molecular Biology;2024-08-02

4. In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord;Molecular Therapy;2024-08

5. Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects;Biomedicines;2024-07-09

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