Timing of Gene Therapy Interventions: The Earlier, the Better
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference15 articles.
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3. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery;Fu;Mol Ther Methods Clin Dev,2016
4. Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: diagnostic, clinical, and biological implications;Yogalingam;Hum Mutat,2001
5. Sanfilippo syndrome: a mini-review;Valstar;J Inherit Metab Dis,2008
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