Zaharieva, I., Cirak, S., Anthony, K., Feng, L., Tasca, G., Ferlini, A., Morgan, J. and Muntoni, F. (2015) G.P.228 - Micro RNA profile associated with the dystrophin level in Becker muscular dystrophy. Neuromuscular Disorders. 25(Supp 2), S255. 0960-8966.
- Information
Information
Abstract:
Becker (BMD) and Duchenne muscular dystrophy (DMD) are allelic disorders arising from mutations in the dystrophin gene. In-frame mutations lead to the milder BMD while out-of-frame mutations disrupt the reading frame and lead to the severe DMD with lack of dystrophin. A therapeutic strategy for skipping specific exons in dystrophin and restoring the open reading frame has been successfully applied in DMD; this “converts” the out-of-frame deletion in DMD to BMD-like in-frame deletion. Micro RNAs (miRs) are small RNA sequences that regulate gene expression post-transcriptionally.
Subjects:
Creators:
Zaharieva, I., Cirak, S., Anthony, K., Feng, L., Tasca, G., Ferlini, A., Morgan, J. and Muntoni, F.
Publisher:
Elsevier
Faculties, Divisions and Institutes:
Date:
October 2015
Date Type:
Publication
Page Range:
S255
Journal or Publication Title:
Neuromuscular Disorders
Volume:
25
Number:
Supp 2
Language:
English
ISSN:
0960-8966
Status:
Published / Disseminated
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