Gene therapy for neurological disorders: progress and prospects-Reference-Cited by-同舟云学术

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Gene therapy for neurological disorders: progress and prospects

Published:2018-08-10 Issue:9 Volume:17 Page:641-659
ISSN:1474-1776
Container-title:Nature Reviews Drug Discovery
language:en
Short-container-title:Nat Rev Drug Discov

Author:

Deverman Benjamin E.,Ravina Bernard M.,Bankiewicz Krystof S.,Paul Steven M.,Sah Dinah W. Y.

Publisher

Springer Science and Business Media LLC

Subject

Drug Discovery,Pharmacology,General Medicine

Link

http://www.nature.com/articles/nrd.2018.110.pdf

Reference249 articles.

1. Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017). This seminal clinical study demonstrates the safety and early efficacy of the first intravenously administered AAV-based therapy designed to correct the SMN deficiency in the CNS.

2. Dominguez, E. et al. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum. Mol. Genet. 20, 681–693 (2011).

3. Foust, K. D. et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat. Biotechnol. 28, 271–274 (2010).

4. Thomsen, G. M. et al. Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. J. Neurosci. 34, 15587–15600 (2014).

5. Borel, F. et al. Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1(G93A) mice and nonhuman primates. Hum. Gene Ther. 27, 19–31 (2016).

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