Dendrimer‐Based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I
Abstract
mRNA‐mediated protein replacement represents a promising concept for the treatment of liver disorders. Children born with fumarylacetoacetate hydrolase (FAH) mutations suffer from Hepatorenal Tyrosinemia Type 1 (HT‐1) resulting in renal dysfunction, liver failure, neurological impairments, and cancer. Protein replacement therapy using FAH mRNA offers tremendous potential to cure HT‐1, but is currently hindered by the development of effective mRNA carriers that can function in diseased livers. Structure‐guided, rational optimization of 5A2‐SC8 mRNA‐loaded dendrimer lipid nanoparticles (mDLNPs) increases delivery potency of FAH mRNA, resulting in functional FAH protein and sustained normalization of body weight and liver function in FAH −/− knockout mice. Optimization using luciferase mRNA produces DLNP carriers that are efficacious at mRNA doses as low as 0.05 mg kg −1 in vivo. mDLNPs transfect > 44% of all hepatocytes in the liver, yield high FAH protein levels (0.5 mg kg −1 mRNA), and are well tolerated in a knockout mouse model with compromised liver function. Genetically engineered FAH −/− mice treated with FAH mRNA mDLNPs have statistically equivalent levels of TBIL, ALT, and AST compared to wild type C57BL/6 mice and maintain normal weight throughout the month‐long course of treatment. This study provides a framework for the rational optimization of LNPs to improve delivery of mRNA broadly and introduces a specific and viable DLNP carrier with translational potential to treat genetic diseases of the liver.
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The therapeutic potential of mRNA-based protein replacement therapies has been tested in other metabolic disorders in preclinical studies, including hereditary tyrosinaemia type I, acute intermittent porphyria, Fabry disease, Crigler-Najjar syndrome type 1, α1 antitrypsin deficiency, methylmalonic acidaemia/aciduria, arginase deficiency, citrin deficiency and glycogen storage disease type I.
“The therapeutic potential of mRNA-based protein replacement therapies has also been tested in other metabolic disorders in preclinical studies, including hereditary tyrosinaemia type I (HTI)142, acute intermittent porphyria222,223, Fabry disease224,225, Crigler–Najjar syndrome type 1 (ref.226), α1 antitrypsin deficiency227, methylmalonic acidaemia/aciduria228, arginase deficiency229, citrin defic”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [142]
In Fah knockout mice i.v. administered hFAH mRNA loaded DLNPs every 3 days for a month, hFAH expression was elevated in the liver.
“The mice were i.v. administrated with hFAH mRNA loaded DLNPs every 3 days for a month, during which hFAH expression was elevated in the liver.[162]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [162]
Long-term use of nitisinone increases the risks of developing hepatic neoplasms and liver failure.
“However, long-term use of nitisinone increases the risks of developing hepatic neoplasms and liver failure.[162]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [162]
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Self-citation
- Authors
- Cheng, Q., Wei, T., Jia, Y., Farbiak, L., Zhou, K., Zhang, S., Wei, Y., Zhu, H., & Siegwart, D. J.
- Affiliations
- Harold C. Simmons Comprehensive Cancer Center (US)
- The University of Texas Southwestern Medical Center (US)
- Corresponding author
- Siegwart, Daniel J.
- ORCID
- Venue
- Advanced Materials
- Publisher
- Wiley
- Published
- 2018-10-25
- Volume
- 30
- Issue
- 52
- Pages
- e1805308-e1805308
- ISSN
- 0935-9648 (ISSN-L)
- 1521-4095
- OpenAlex
- W2898487406
- Cited by
- 221
- References
- 49
- Citations by year
- 2026: 16 · 2025: 30 · 2024: 36 · 2023: 38 · 2022: 32 · 2021: 30 · 2020: 26 · 2019: 13
- FWCI
- 6.13
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- Advanced Materials (publishedVersion)
- PubMed (publishedVersion)
- Funders
- U.S. Department of Defense (CA150245P3)
- American Cancer Society (RSG‐17‐012‐01)
- Cancer Prevention and Research Institute of Texas (R1212)
- Welch Foundation (I‐1855)
- National Institutes of Health (R01CA190525)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- fumarylacetoacetate hydrolase, mRNA delivery, liver-directed gene therapy, hepatocyte transfection, hereditary liver disease
- MeSH terms
- Animals, Disease Models, Animal, Hydrolases, Liver, Mice, Inbred C57BL, Mice, Transgenic, Random Allocation, RNA, Messenger, Genetic Therapy, Tyrosinemias, Dendrimers, Nanoparticles
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