Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy-Reference-Cited by-同舟云学术

Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy

Published:2015-09 Issue:1-2 Volume:116 Page:80-87
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Gray-Edwards Heather L.,Brunson Brandon L.,Holland Merrilee,Hespel Adrien-Maxence,Bradbury Allison M.,McCurdy Victoria J.,Beadlescomb Patricia M.,Randle Ashley N.,Salibi Nouha,Denney Thomas S.,Beyers Ronald J.,Johnson Aime K.,Voyles Meredith L.,Montgomery Ronald D.,Wilson Diane U.,Hudson Judith A.,Cox Nancy R.,Baker Henry J.,Sena-Esteves Miguel,Martin Douglas R.

Funder

NIH

National Tay–Sachs and Allied Diseases

Cure Tay–Sachs Foundation

Scott-Ritchey Research Center

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

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4. Cardiac involvement in Sandhoff's disease. Inborn error of glycosphingolipid metabolism;Blieden;Am. J. Cardiol.,1974

5. Therapeutic response in feline Sandhoff disease despite immunity to intracranial gene therapy;Bradbury;Mol. Ther.,2013

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