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...

Celý popis

Uloženo v:
Podrobná bibliografie
Hlavní autoři: Glascock, Jacqueline J., Osman, Erkan Y., Wetz, Mary J., Krogman, Megan M., Shababi, Monir, Lorson, Christian L.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2012
Témata:
Brief Report
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky