Gene Therapy for Pompe Disease: The Time is now
Author:
Affiliation:
1. Genethon, Evry, France
2. Department of Pediatrics, Stanford University, Stanford, California
3. Spark Therapeutics, Philadelphia, Pennsylvania.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.109
Reference191 articles.
1. Pompe's disease
2. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease
3. Childhood Pompe disease: clinical spectrum and genotype in 31 patients
4. The emerging phenotype of late-onset Pompe disease: A systematic literature review
5. Neural deficits contribute to respiratory insufficiency in Pompe disease
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