AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study-Reference-Cited by-同舟云学术

AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

Published:2021-12 Issue: Volume:23 Page:644-658
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Hu Huimin,Mosca Rosario,Gomero Elida,van de Vlekkert Diantha^ORCID,Campos Yvan,Fremuth Leigh E.^ORCID,Brown Scott A.^ORCID,Weesner Jason A.^ORCID,Annunziata Ida^ORCID,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

Reference44 articles.

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

2. Galactosialidosis, 8th edition;d'Azzo,2013

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

4. Pathogenesis, emerging therapeutic targets and treatment in sialidosis;d'Azzo;Expert Opin. Orphan Drugs,2015

5. Mouse model for the lysosomal disorder galactosialidosis and correction of the phenotype with over-expressing erythroid precursor cells;Zhou;Genes Dev.,1995

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