Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders-Reference-Cited by-同舟云学术

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Published:2011-11 Issue:11 Volume:19 Page:1971-1980
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Bevan Adam K,Duque Sandra,Foust Kevin D,Morales Pablo R,Braun Lyndsey,Schmelzer Leah,Chan Curtis M,McCrate Mary,Chicoine Louis G,Coley Brian D,Porensky Paul N,Kolb Stephen J,Mendell Jerry R,Burghes Arthur HM,Kaspar Brian K

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference50 articles.

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2. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice;Dominguez;Hum Mol Genet,2011

3. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN;Foust;Nat Biotechnol,2010

4. Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy;Valori;Sci Transl Med,2010

5. Survival of motor neuron protein over-expression prevents calpain-mediated cleavage and activation of procaspase-3 in differentiated human SH-SY5Y cells;Anderton;Neuroscience,2011

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