Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy-Reference-Cited by-同舟云学术

Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy

Published:2021-06-07 Issue:4 Volume:35 Page:389-399
ISSN:1173-8804
Container-title:BioDrugs
language:en
Short-container-title:BioDrugs

Author:

Sheikh Omar,Yokota Toshifumi^ORCID

Funder

WCHRI

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Pharmacology,General Medicine,Biotechnology

Link

https://link.springer.com/content/pdf/10.1007/s40259-021-00486-7.pdf

Reference130 articles.

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2. Li D, Mastaglia FL, Fletcher S, Wilton SD. Precision medicine through antisense oligonucleotide-mediated exon skipping. Trends Pharmacol Sci. 2018;39(11):982–94.

3. Duchenne DR. The Pathology of paralysis with muscular degeneration (paralysie myosclerotique), or paralysis with apparent hypertrophy. Br Med J. 1867;2:541–2.

4. Moat SJ, Bradley DM, Salmon R, Clarke A, Hartley L. Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK). Eur J Hum Genet. 2013;21(10):1049–53.

5. Worton RG. Duchenne muscular dystrophy: gene and gene product; mechanism of mutation in the gene. J Inherit Metab Dis. 1992;15(4):539–50.

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