Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference227 articles.
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2. Lysosomal storage diseases;Platt;Nat. Rev. Dis. Prim.,2018
3. Fabry disease;Germain;Orphanet J. Rare Dis.,2010
4. Life expectancy and cause of death in males and females with Fabry disease: findings from the Fabry Registry;Waldek;Genet. Med.,2009
5. Early initiation of enzyme replacement therapy in classical Fabry disease normalizes biomarkers in clinically asymptomatic pediatric patients;Kritzer;Mol. Genet. Metab. Rep.,2019
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