In VivoGene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
Author:
Affiliation:
1. Center of Animal Biotechnology and Gene Therapy and Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona (UAB), Bellaterra, Spain
2. CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.217
Reference126 articles.
1. 1. NeufeldEF, MuenzerJ. The mucopolysaccharidoses. In: SchriverC, BeaudetA, SlyW, et al., eds. Metabolic and Molecular Bases of Inherited Disease. New York, McGraw-Hill, 2001:3421–3452.
2. Lysosomal Storage Diseases: From Pathophysiology to Therapy
3. Sanfilippo syndrome: causes, consequences, and treatments
4. Sanfilippo syndrome: A mini-review
5. The Sanfilippo A Corrective Factor
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