Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

Author:

McCurdy Victoria J.,Johnson Aime K.,Gray-Edwards Heather L.,Randle Ashley N.,Bradbury Allison M.,Morrison Nancy E.,Hwang Misako,Baker Henry J.,Cox Nancy R.,Sena-Esteves Miguel,Martin Douglas R.ORCID

Funder

U.S. Department of Health & Human Services | National Institutes of Health

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference52 articles.

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2. Conzelmann E, Kytzia H, Navon R, Sandhoff K. Ganglioside GM2 N-acetyl-beta-D-galactosaminidase activity in cultured fibroblasts of late-infantile and adult GM2 gangliosidosis patients and of healthy probands with low hexosaminidase level. Am J Hum Genet. 1983;35:900–14.

3. Jacobs J, Willemsen M, Groot-Loonen J, Wevers R, Hoogerbrugge P. Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease. Bone Marrow Transplant. 2005;36:925–6.

4. Bembi B, Marchetti F, Guerci V, Ciana G, Addobbati R, Grasso D, et al. Substrate reduction therapy in the infantile form of Tay-Sachs disease. Neurology. 2006;66:278–80.

5. Shapiro BE, Pastores GM, Gianutsos J, Luzy C, Kolodny EH. Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment. Genet Med. 2009;11:425–33.

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