Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia.

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Bibliographic Details
Title: Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia.
Authors: May FJ; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, MD, USA.; NIH Medical Research Scholars Program, Bethesda, MD, USA.; Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, Cleveland, OH, USA., Head PE; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, MD, USA., Venturoni LE; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, MD, USA., Chandler RJ; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, MD, USA., Venditti CP; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, MD, USA.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 Jun 03; Vol. 21, pp. 765-776. Date of Electronic Publication: 2021 Jun 03 (Print Publication: 2021).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2021.04.005