Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Editorial
. 2017 Aug;10(8):293-296.
doi: 10.1177/1756285617717155. Epub 2017 Jun 28.

Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost

Marinos C Dalakas  1
Affiliations
Editorial

Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost

Marinos C Dalakas. Ther Adv Neurol Disord. 2017 Aug.
No abstract available

PubMed Disclaimer

Conflict of interest statement

Conflict of interest statement: The authors declare that there is no conflict of interest.

References

    1. Bushby K, Finkel R, Birnkrant DJ, et al. ; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9: 77–93. - PubMed
    1. Ervasti JM, Campbell KP. A role for the dystrophin–glycoprotein complex as a transmembrane linker between laminin and actin. J Cell Biol 1993; 122: 809–823. - PMC - PubMed
    1. Lai Y, Yue Y, Liu M, et al. Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol 2005; 23: 1435. - PMC - PubMed
    1. Ramos J, Chamberlain JS. Gene therapy for Duchenne muscular dystrophy. Expert Opin Orphan Drugs 2015; 3: 1255–1266. - PMC - PubMed
    1. Reining AM, Mizraei S, Berlau DJ. Advances in the treatment of Duchenne muscular dystrophy. Ther Rev 2016; 37(4): 492–499. - PubMed

Publication types

LinkOut - more resources