Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease-Reference-Cited by-同舟云学术

Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Published:2016-07 Issue:7 Volume:27 Page:509-521
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Karumuthil-Melethil Subha¹,Nagabhushan Kalburgi Sahana¹,Thompson Patrick²,Tropak Michael³,Kaytor Michael D.⁴,Keimel John G.⁴,Mark Brian L.⁵,Mahuran Don³⁶,Walia Jagdeep S.²,Gray Steven J.¹⁷

Affiliation:

1. Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

2. Medical Genetics/Departments of Pediatrics, Queen's University, Kingston, Ontario, Canada.

3. Genetics and Genome Biology, SickKids, Toronto, Ontario, Canada.

4. New Hope Research Foundation, North Oaks, Minnesota.

5. Department of Microbiology, University of Manitoba, Winnipeg, Manitoba, Canada.

6. Department of Laboratory Medicine and Pathology, University of Toronto, Toronto, Ontario, Canada.

7. Department of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.013

Reference52 articles.

1. Biochemistry and Genetics of gangliosidoses

2. Quantitative correlation between the residual activity of β-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

3. Targeted disruption of the Hexa gene results in mice with biochemical and pathologic features of Tay-Sachs disease.

4. Mouse models of Tay–Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism

5. Dramatically Different Phenotypes in Mouse Models of Human Tay-Sachs and Sandhoff Diseases

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