Loading...

Decreasing Disease Severity in Symptomatic, Smn(−/−);SMN2(+/+), Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the homozygous loss of Survival Motor Neuron-1 (SMN1). In humans, a nearly identical copy gene is present, SMN2. SMN2 is retained in all SMA patients and e...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Main Authors: Glascock, Jacqueline J., Osman, Erkan Y., Wetz, Mary J., Krogman, Megan M., Shababi, Monir, Lorson, Christian L.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2012
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3300100/
https://ncbi.nlm.nih.gov/pubmed/22029744
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.166
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!