A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy

Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. However, in vivo studies validating the effectiveness of a short ASO (<10-mer) in the context of a human disease have not been performed. One dis...

Celý popis

Uloženo v:
Podrobná bibliografie
Hlavní autoři: Jeffrey M Keil, Joonbae Seo, Matthew D Howell, Walter H Hsu, Ravindra N Singh, Christine J DiDonato
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2014-01-01
Edice:Molecular Therapy: Nucleic Acids
Témata:
antisense oligonucleotide
cardiac
short ASO
skeletal muscle
SMA mouse model
SMN2
spinal muscular atrophy
splicing
On-line přístup:http://www.sciencedirect.com/science/article/pii/S2162253116303122
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky