NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice

Spinal muscular atrophy (SMA) is a neuromuscular disease causing the most frequent genetic childhood lethality. Recently, nusinersen, an antisense oligonucleotide (ASO) that corrects SMN2 splicing and thereby increases full-length SMN protein, has been approved by the FDA and EMA for SMA therapy. Ho...

Full description

Saved in:
Bibliographic Details
Published in:Am J Hum Genet
Main Authors: Torres-Benito, Laura, Schneider, Svenja, Rombo, Roman, Ling, Karen K., Grysko, Vanessa, Upadhyay, Aaradhita, Kononenko, Natalia L., Rigo, Frank, Bennett, C. Frank, Wirth, Brunhilde
Format: Artigo
Language:Inglês
Published: Elsevier 2019
Subjects:
Report
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6612520/
https://ncbi.nlm.nih.gov/pubmed/31230718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2019.05.008
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items