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

作者：Randy J. Chandler, Charles P. Venditti · 发表于：Human Gene Therapy · 年份：2019 · DOI：10.1089/hum.2019.113 · 被引用次数：56 · 研究领域：Virus-based gene therapy research、Metabolism and Genetic Disorders、Mitochondrial Function and Pathology

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.