Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis-Reference-Cited by-同舟云学术

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Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis

Published:2013-10 Issue:10 Volume:1832 Page:1784-1792
ISSN:0925-4439
Container-title:Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
language:en
Short-container-title:Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Author:

Bonten Erik J.,Yogalingam Gouri,Hu Huimin,Gomero Elida,van de Vlekkert Diantha,d'Azzo Alessandra

Funder

National Institutes of Health

Assisi Foundation of Memphis

American Lebanese Syrian Associated Charities (ALSAC)

Publisher

Elsevier BV

Subject

Molecular Biology,Molecular Medicine

Reference41 articles.

1. Transport of human lysosomal neuraminidase to mature lysosomes requires protective protein/cathepsin A;van der Spoel;EMBO J.,1998

2. Lysosomal neuraminidase. Catalytic activation in insect cells is controlled by the protective protein/cathepsin A;Bonten;J. Biol. Chem.,2000

3. Heterodimerization of the sialidase NEU1 with the chaperone protective protein/cathepsin A prevents its premature oligomerization;Bonten;J. Biol. Chem.,2009

4. Characterization of human lysosomal neuraminidase defines the molecular basis of the metabolic storage disorder sialidosis;Bonten;Genes Dev.,1996

5. Novel mutations in lysosomal neuraminidase identify functional domains and determine clinical severity in sialidosis;Bonten;Hum. Mol. Genet.,2000

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