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
. 2013 Mar;21(3):505-6.
doi: 10.1038/mt.2013.25.

Gene therapy for Canavan's disease takes a step forward

Seemin S Ahmed  1 , Guangping Gao
Affiliations

Gene therapy for Canavan's disease takes a step forward

Seemin S Ahmed et al. Mol Ther. 2013 Mar.
No abstract available

PubMed Disclaimer

References

    1. Matalon R, Michals K, Sebesta D, Deanching M, Gashkoff P., and, Casanova J. Aspartoacylase deficiency and N-acetylaspartic aciduria in patients with Canavan disease. Am J Med Genet. 1988;29:463–471. - PubMed
    1. Stenson PD, Ball EV, Mort M, Phillips AD, Shiel JA, Thomas NS.et al. (2003Human Gene Mutation Database (HGMD): 2003 update Hum Mutat 21577–581. - PubMed
    1. Matalon R, Kaul R, Gao GP, Michals K, Gray RG, Bennett-Briton S.et al. (1995Prenatal diagnosis for Canavan disease: the use of DNA markers J Inherit Metab Dis 18215–217. - PubMed
    1. Kaul R, Gao GP, Balamurugan K., and, Matalon R. Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease. Nat Genet. 1993;5:118–123. - PubMed
    1. Matalon R., and, Michals-Matalon K. Molecular basis of Canavan disease. Eur J Paediatr Neurol. 1998;2:69–76. - PubMed