ReviewWiley interdisciplinary reviews. Nanomedicine and nanobiotechnology
Therapeutic Application of mRNA for Genetic Diseases.
Review in Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
3 citing papers in PubMed.
- mRNA Therapeutics Beyond Infectious Diseases: Expanding Therapeutic Applications and Future Perspectives.Immunity, inflammation and disease · 2026Review
- Next-Generation Nanocarrier Platforms for RNA Vaccines: Advances in Formulation, Stability Engineering, and Translational Manufacturing Challenges.Pharmaceutics · 2026Review
- Removal of Toxic Metabolites-Chelation: Manganese Disorders.Journal of inherited metabolic disease · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
While gene therapy has been at the center of an active research field for decades, messenger RNA (mRNA) has long been considered unsuited for therapeutic application due to challenges with stability, immunogenicity, and delivery. Where gene therapy focuses on providing the desired genetic code, mRNA can directly provide the instructions encoded in the corresponding gene. This review aims to explore recent advances in mRNA therapies, building on the success of mRNA COVID-19 vaccines, and extend these insights to the potential treatment of rare genetic diseases. We follow the "outside-in" trajectory of mRNA therapies from administration to intracellular function, focusing on carrier systems such as lipid nanoparticles and virus-like particles, mRNA modifications, and the potential and challenges for clinical applications. To treat rare diseases, different approaches can be envisioned, including chronic or acute delivery of mRNAs encoding functional enzymes for enzyme deficiencies and delivery of CRISPR/Cas9-based gene-editing tools for gene correction. These different approaches determine safety and immunological considerations. By exploring genetic, technical, and therapeutic aspects, this review highlights the potential and current challenges of mRNA therapies to address the large unmet needs in rare genetic disorders.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.