Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Loss of Survival Motor Neuron-1 (SMN1) causes Spinal Muscular Atrophy, a devastating neurodegenerative disease. SMN2 is a nearly identical copy gene; however SMN2 cannot prevent disease development in the absence of SMN1 since the majority of SMN2-derived transcripts are alternatively spliced, encod...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Gepubliceerd in:Mol Ther
Hoofdauteurs: Osman, Erkan Y, Washington, Charles W, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian L
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2016
Onderwerpen:
Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5113110/
https://ncbi.nlm.nih.gov/pubmed/27401142
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.145
Tags: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items