In Cellulo Examination of a Beta-Alpha Hybrid Construct of Beta-Hexosaminidase A Subunits, Reported to Interact with the GM2 Activator Protein and Hydrolyze GM2 Ganglioside-Reference-Cited by-同舟云学术

In Cellulo Examination of a Beta-Alpha Hybrid Construct of Beta-Hexosaminidase A Subunits, Reported to Interact with the GM2 Activator Protein and Hydrolyze GM2 Ganglioside

Published:2013-03-04 Issue:3 Volume:8 Page:e57908
ISSN:1932-6203
Container-title:PLoS ONE
language:en
Short-container-title:PLoS ONE

Author:

Sinici Incilay,Yonekawa Sayuri,Tkachyova Ilona,Gray Steven J.,Samulski R. Jude,Wakarchuk Warren,Mark Brian L.,Mahuran Don J.

Publisher

Public Library of Science (PLoS)

Subject

Multidisciplinary

Reference40 articles.

1. The Biochemistry of HEXA and HEXB Gene Mutations Causing GM2 Gangliosidosis;DJ Mahuran;Biochim Biophys Acta,1991

2. Crystal structure of human beta-hexosaminidase B: understanding the molecular basis of Sandhoff and Tay-Sachs disease;BL Mark;J Mol Biol,2003

3. The X-ray crystal structure of human beta-hexosaminidase B provides new insights into Sandhoff disease;T Maier;J Mol Biol,2003

4. Crystallographic Structure of Human beta-Hexosaminidase A: Interpretation of Tay-Sachs Mutations and Loss of G(M2) Ganglioside Hydrolysis;MJ Lemieux;J Mol Biol,2006

5. Crystal structure of human GM2-activator protein with a novel beta-cup topology;CS Wright;J Mol Biol,2000

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