Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?-Reference-Cited by-同舟云学术

Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?

Published:2014-04-02 Issue:230 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Hoffman Eric P.¹,McNally Elizabeth M.²

Affiliation:

1. Center for Genetic Medicine Research, Children’s National Medical Center, 111 Michigan Avenue, NW, Washington, DC 20010, USA.

2. Departments of Medicine and Human Genetics, Institute for Cardiovascular Research, University of Chicago Medical Center, 5841 S. Maryland Avenue, MC 6088, Chicago, IL 60637, USA.

Abstract

Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage de-risking and regulatory guidance.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference9 articles.

1. Exon skipping during splicing of dystrophin mRNA precursor due to an intraexon deletion in the dystrophin gene of Duchenne muscular dystrophy kobe.

2. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

3. Dystrophin expression in themdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

4. Dosing Regimen Has a Significant Impact on the Efficiency of Morpholino Oligomer-Induced Exon Skipping in mdx Mice

5. Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

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