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
Comment
. 2015 Apr;23(4):614-5.
doi: 10.1038/mt.2015.38.

A shot in the bone corrects a genetic disease

Affiliations
Comment

A shot in the bone corrects a genetic disease

Brian D Brown. Mol Ther. 2015 Apr.
No abstract available

PubMed Disclaimer

Comment on

References

    1. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L.et al. (2009Gene therapy for immunodeficiency due to adenosine deaminase deficiency N Engl J Med 360447–458. - PubMed
    1. Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T.et al. (2013Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy Science 3411233158. - PubMed
    1. Naldini L. Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet. 2011;12:301–315. - PubMed
    1. Chuah MK, Evens H., and, VandenDriessche T. Gene therapy for hemophilia. J Thromb Haemost. 2013;11 suppl. 1:99–110. - PubMed
    1. Moayeri M, Ramezani A, Morgan RA, Hawley TS., and, Hawley RG. Sustained phenotypic correction of hemophilia A mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells. Mol Ther. 2004;10:892–902. - PubMed