Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
Open accessAbstract
Isolated methylmalonic acidemia/aciduria (MMA) is a devastating metabolic disorder with poor outcomes despite current medical treatments. Like other mitochondrial enzymopathies, enzyme replacement therapy (ERT) is not available, and although promising, AAV gene therapy can be limited by pre-existing immunity and has been associated with genotoxicity in mice. To develop a new class of therapy for MMA, we generated a pseudoU-modified codon-optimized mRNA encoding human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, and encapsulated it into biodegradable lipid nanoparticles (LNPs). Intravenous (i.v.) administration of hMUT mRNA in two different mouse models of MMA resulted in a 75%-85% reduction in plasma methylmalonic acid and was associated with increased hMUT protein expression and activity in liver. Repeat dosing of hMUT mRNA reduced circulating metabolites and dramatically improved survival and weight gain. Additionally, repeat i.v. dosing did not increase markers of liver toxicity or inflammation in heterozygote MMA mice.
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Supplement of desired enzymes by lipid nanoparticle-mRNA formulations can slow down disease progression.
“Therefore, the supplement of desired enzymes by lipid nanoparticle–mRNA formulations can slow down disease progression42,221.”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [221]
Repeated administrations of hMUT mRNA improved growth and survival of MMA mice without obvious liver damage and inflammation.
“In addition, repeated administrations improved the growth and survival of MMA mice without obvious liver damage and inflammation.[47]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [47]
Systemic injection of hMUT mRNA formulated with MC3 lipid demonstrated rapid (as early as 2 h) and effective (maximum 90% at 24 h) reduction of plasma methylmalonic acid.
“Systemic injection of human MUT (hMUT) mRNA, formulated with MC3 lipid, demonstrated a rapid (as early as 2 h after administration) and effective (maximum 90% at 24 h) reduction of plasma methylmalonic acid.[47]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [47]
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Self-citation
- Authors
- An, D., Schneller, J. L., Frassetto, A., Liang, S., Zhu, X., Park, J.-S., Theisen, M., Hong, S.-J., Zhou, J., Rajendran, R., Levy, B., Howell, R., Besin, G., Presnyak, V., Sabnis, S., Murphy-Benenato, K. E., Kumarasinghe, E. S., Salerno, T., Mihai, C., Lukacs, C. M., Chandler, R. J., Guey, L. T., Venditti, C. P., & Martini, P. G. V.
- Affiliations
- Moderna Therapeutics (United States) (US)
- National Institutes of Health (US)
- Corresponding author
- Venditti, Charles P.
- Martini, Paolo G.V.
- ORCID
- Venue
- Cell Reports
- Publisher
- Elsevier BV
- Published
- 2017-12
- Volume
- 21
- Issue
- 12
- Pages
- 3548-3558
- ISSN
- 2211-1247 (ISSN-L)
- PMC
- PMC9667413
- OpenAlex
- W2777634859
- Cited by
- 263
- References
- 55
- Citations by year
- 2026: 22 · 2025: 29 · 2024: 34 · 2023: 35 · 2022: 45 · 2021: 33 · 2020: 19 · 2019: 32 · 2018: 13 · 2014: 1
- FWCI
- 7.15
- Type
- journal article
- Language
- en
- Update notices
- erratum (2018-08-28)
- Open access
- gold
- doi.org
- Licence
- Locations
- Cell Reports (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion, cc-by-nc-nd)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Funders
- National Human Genome Research Institute
- National Institutes of Health
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Keywords
- methylmalonic acidemia, methylmalonyl-CoA mutase, mRNA therapeutics, lipid nanoparticles, hepatotoxicity
- MeSH terms
- Amino Acid Metabolism, Inborn Errors, Animals, Female, Humans, Lipids, Liver, Male, Methylmalonyl-CoA Mutase, RNA, Messenger, Genetic Therapy, Mice, Nanoparticles, Administration, Intravenous
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