ArticleScientific reports2018
mRNA treatment produces sustained expression of enzymatically active human ADAMTS13 in mice.
Article in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 20 citations in OpenAlex.
- Long Term Follow Up of Hereditary Thrombotic Thrombocytopenic Purpura on Plasma Therapy for 9 Years: A Case Report.Clinical case reports · 2026Article
- The Nucleolus in Human Disease: Ribosome Biogenesis, Nucleolar Surveillance, and Therapeutic Opportunities.Biomolecules · 2026Review
- Thrombotic Thrombocytopenic Purpura in Pediatric Patients.Biomedicines · 2025Review
- Novel mechanisms of action of emerging therapies of hereditary thrombotic thrombocytopenic purpura.Expert review of hematology · 2024Review
- Data-balanced transformer for accelerated ionizable lipid nanoparticles screening in mRNA delivery.Briefings in bioinformatics · 2024Article
- Nanomaterials for mRNA-based therapeutics: Challenges and opportunities.Bioengineering & translational medicine · 2023Review
- mRNA nanomedicine: Design and recent applications.Exploration (Beijing, China) · 2022Review
- TTP: From empiricism for an enigmatic disease to targeted molecular therapies.British journal of haematology · 2022Review
- Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety.Biomedicines · 2021Review
- Lipid nanoparticles for mRNA delivery.Nature reviews. Materials · 2021Review
- Optimization of 5' Untranslated Region of Modified mRNA for Use in Cardiac or Hepatic Ischemic Injury.Molecular therapy. Methods & clinical development · 2020Article
- [Advances in the treatment of thrombotic thrombocytopenic purpura].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2019Article
- RNA delivery biomaterials for the treatment of genetic and rare diseases.Biomaterials · 2019Review
- Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Review
- mRNA as novel technology for passive immunotherapy.Cellular and molecular life sciences : CMLS · 2019Review
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Thrombotic thrombocytopenic purpura (TTP) is primarily caused by deficiency of ADAMTS13 within the blood stream due to either genetic defects or presence of inhibitory autoantibodies. Preclinical and clinical studies suggest that enzyme replacement therapy with recombinant human ADAMTS13 protein (rhADAMTS13) is effective and safe in treatment of TTP. However, frequent dosing would be required due to the relatively short half-life of rhADAMTS13 in circulation as well as the presence of inhibitory autoantibodies that collectively result in the poor pharmacological profile of rhADAMTS13. With technical breakthroughs in exploring mRNA as therapeutics, we hypothesized that restoration of ADAMTS13 activity for a prolonged duration of time can be achieved through systemic dosing of mRNA, wherein the dosed mRNA would utilize hepatic cells as bioreactors for continuous production of ADAMTS13. To test this hypothesis, mRNA encoding human ADAMTS13 WT or an ADAMTS13 variant, that had demonstrated resistance to predominant clinical TTP autoantibodies, was formulated in lipid nano-particles for liver-targeted delivery. In both ADAMTS13-sufficient and -deficient mice, a single dose of the formulated mRNAs at 1 mg/kg resulted in expression of hADAMTS13 at or above therapeutically relevant levels in mice for up to five days. This proof-of-concept study suggests that mRNA therapy could provide a novel approach for TTP treatment.
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