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Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future
Randy J Chandler1, Charles P Venditti1
1Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland.
Human Gene Therapy
|July 16, 2019
Summary
New genomic therapies show promise for treating methylmalonic acidemia (MMA), a severe metabolic disorder. This review covers viral gene therapy and newer approaches for MMA and related organic acid metabolism disorders.
Area of Science:
- Biochemistry and Genetics
- Metabolic Disorders
- Gene Therapy
Background:
- Methylmalonic acidemia (MMA) is a severe, life-threatening monogenic metabolic disorder.
- Current treatments for MMA are limited, necessitating the development of novel therapeutic strategies.
- Genetic disorders like MMA are increasingly being targeted by advanced genomic therapies.
Purpose of the Study:
- To review traditional viral gene therapy approaches for methylmalonic acidemia (MMA).
- To explore promising newer genomic therapies for MMA, including gene editing and mRNA therapy.
- To discuss the potential of these therapies for treating MMA and related organic acid metabolism disorders.
Main Methods:
- Review of existing literature on viral gene therapy in animal models of MMA.
- Analysis of emerging genomic therapeutic modalities such as adeno-associated virus gene addition and genome editing.
- Evaluation of systemic mRNA therapy as a potential treatment for MMA.
Main Results:
- Traditional viral gene therapy has provided proof-of-concept in preclinical MMA models.
- Newer genomic therapies, including gene editing and mRNA therapy, demonstrate significant promise in murine models of MMA.
- Each therapeutic approach possesses distinct advantages and disadvantages for genetic disorder treatment.
Conclusions:
- Genomic therapies offer a promising future for treating methylmalonic acidemia (MMA).
- Further research into gene addition, genome editing, and mRNA therapy is warranted for MMA and related metabolic disorders.
- These advanced therapeutic strategies may lead to improved clinical outcomes for patients with organic acidemias.
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