Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice-Reference-Cited by-同舟云学术

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

Published:2017-06-07 Issue:1 Volume:8 Page:
ISSN:2041-1723
Container-title:Nature Communications
language:en
Short-container-title:Nat Commun

Author:

Tasfaout Hichem,Buono Suzie,Guo Shuling,Kretz Christine,Messaddeq Nadia,Booten Sheri,Greenlee Sarah,Monia Brett P.,Cowling Belinda S.^ORCID,Laporte Jocelyn

Publisher

Springer Science and Business Media LLC

Subject

General Physics and Astronomy,General Biochemistry, Genetics and Molecular Biology,General Chemistry

Link

http://www.nature.com/articles/ncomms15661.pdf

Reference30 articles.

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3. Wheeler, T. M. et al. Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature 488, 111–115 (2012).

4. Finkel, R. S. et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet 388, 3017–3026 (2016).

5. Ravenscroft, G. et al. Pathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focus. Brain 138, 246–268 (2015).

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