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Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Authors
  • Chandler, Randy J1
  • Venditti, Charles P1
  • 1 Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland.
Type
Published Article
Journal
Human Gene Therapy
Publisher
Mary Ann Liebert
Publication Date
Oct 01, 2019
Volume
30
Issue
10
Pages
1236–1244
Identifiers
DOI: 10.1089/hum.2019.113
PMID: 31303064
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Methylmalonic acidemia (MMA) is a severe, and sometimes lethal, monogenic metabolic disorder in need of improved treatments. A number of new genomic therapies, which include canonical adeno-associated virus gene addition, genome editing, and systemic mRNA therapy, have shown great promise in murine models of MMA. Each approach has unique advantages and disadvantages for treating genetic disorders like MMA. This article reviews traditional viral gene therapy experiments that have provided enabling proof of concept studies in animal models, and newer approaches that may emerge as effective treatments for MMA and related disorders of organic acid metabolism.

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