Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.

Duchenne and Becker muscular dystrophy severity depends upon the nature and location of the DMD gene lesion and generally correlates with the dystrophin open reading frame. However, there are striking exceptions where an in-frame genomic deletion leads to severe pathology or protein-truncating mutat...

Full description

Bibliographic Details
Main Authors: Zhi Yon Charles Toh, May Thandar Aung-Htut, Gavin Pinniger, Abbie M Adams, Sudarsan Krishnaswarmy, Brenda L Wong, Sue Fletcher, Steve D Wilton
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2016-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC4706350?pdf=render