G.P.228 - Micro RNA profile associated with the dystrophin level in Becker muscular dystrophy

I Zaharieva, S Cirak, Karen Anthony, L Feng, G Tasca, A Ferlini, J Morgan, F Muntoni

Research output: Contribution to JournalAbstract

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.
Original languageEnglish
JournalNeuromuscular Disorders
Volume25
Issue numberSupp 2
Publication statusPublished - 1 Oct 2015

Fingerprint Dive into the research topics of 'G.P.228 - Micro RNA profile associated with the dystrophin level in Becker muscular dystrophy'. Together they form a unique fingerprint.

  • Cite this

    Zaharieva, I., Cirak, S., Anthony, K., Feng, L., Tasca, G., Ferlini, A., Morgan, J., & Muntoni, F. (2015). G.P.228 - Micro RNA profile associated with the dystrophin level in Becker muscular dystrophy. Neuromuscular Disorders, 25(Supp 2).