A gene-edited mouse model of limb-girdle muscular dystrophy 2C for testing exon skipping

A gene-edited mouse model of limb-girdle muscular dystrophy 2C for testing exon skipping

Limb-girdle muscular dystrophy type 2C is caused by autosomal recessive mutations in the γ-sarcoglycan (SGCG) gene. The most common SGCG mutation is a single nucleotide deletion from a stretch of five thymine residues in SGCG exon 6 (521ΔT). This founder mutation disrupts the transcript reading fram...

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Bibliographic Details
Main Authors: Alexis R. Demonbreun, Eugene J. Wyatt, Katherine S. Fallon, Claire C. Oosterbaan, Patrick G. Page, Michele Hadhazy, Mattia Quattrocelli, David Y. Barefield, Elizabeth M. McNally
Format: Article
Language:English
Published: The Company of Biologists 2020-02-01
Series:Disease Models & Mechanisms
Subjects:
lgmd 2c
antisense oligonucleotide
sarcoglycan
dystrophin
gene correction
mouse
Online Access:http://dmm.biologists.org/content/13/2/dmm040832

Internet

http://dmm.biologists.org/content/13/2/dmm040832

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