Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept-Reference-Cited by-同舟云学术

Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept

Published:2021-03 Issue: Volume:20 Page:191-203
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Cadaoas Jaclyn,Hu Huimin,Boyle Gabrielle,Gomero Elida,Mosca Rosario,Jayashankar Kartika,Machado Mike,Cullen Sean,Guzman Belle,van de Vlekkert Diantha,Annunziata Ida,Vellard Michel,Kakkis Emil,Koppaka Vish,d’Azzo Alessandra

Funder

American Lebanese Syrian Associated Charities

Ultragenyx Pharmaceutical

National Institutes of Health

Assisi Foundation of Memphis

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference47 articles.

1. Molecular defect in combined beta-galactosidase and neuraminidase deficiency in man;D’Azzo;Proc. Natl. Acad. Sci. USA,1982

2. Lysosomal multienzyme complex: pros and cons of working together;Bonten;Cell. Mol. Life Sci.,2014

3. Galactosialidosis: historic aspects and overview of investigated and emerging treatment options;Annunziata;Expert Opin. Orphan Drugs,2017

4. Galactosialidosis. The Online Metabolic and Molecular Bases of Inherited Disease;d’Azzo,2014

5. Expression of cDNA encoding the human “protective protein” associated with lysosomal beta-galactosidase and neuraminidase: homology to yeast proteases;Galjart;Cell,1988

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