Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy
Author:
Affiliation:
1. Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina
2. Department of Pathology, Sanofi Genzyme, Framingham, Massachusetts.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.099
Reference38 articles.
1. Glycogen storage disease type IV, amylopectinosis.
2. Clinical and genetic heterogeneity of branching enzyme deficiency (glycogenosis type IV)
3. The Variable Presentations of Glycogen Storage Disease Type IV: A Review of Clinical, Enzymatic and Molecular Studies
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