Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

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

19 citing papers in PubMed.

1. mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment.Molecular therapy. Nucleic acids · 2025
   Review
2. Exploring the Potential and Advancements of Circular RNA Therapeutics.Exploration (Beijing, China) · 2025
   Review
3. mRNA therapy as primary and bridge therapy for inborn errors of metabolism.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
   Article
4. Recent Advances and Prospects in RNA Drug Development.International journal of molecular sciences · 2024
   Review
5. Frameworks for transformational breakthroughs in RNA-based medicines.Nature reviews. Drug discovery · 2024
   Review
6. Ultrasound-mediated gene delivery specifically targets liver sinusoidal endothelial cells for sustained FVIII expression in hemophilia A mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
   Article
7. Inborn errors of amino acid metabolism - from underlying pathophysiology to therapeutic advances.Disease models & mechanisms · 2023
   Article
8. Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia.Journal of inherited metabolic disease · 2023
   Review
9. The legacy of mRNA engineering: A lineup of pioneers for the Nobel Prize.Molecular therapy. Nucleic acids · 2022
   Review
10. Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia.Journal of inherited metabolic disease · 2022
    Article
11. Chemically modified mRNA beyond COVID-19: Potential preventive and therapeutic applications for targeting chronic diseases.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2022
    Review
12. Delivery of therapeutic oligonucleotides in nanoscale.Bioactive materials · 2022
    Review
13. Molecular therapy. Methods & clinical development · 2021
    Article
14. Mitochondrial disease, mitophagy, and cellular distress in methylmalonic acidemia.Cellular and molecular life sciences : CMLS · 2021
    Review
15. Modifications in an Emergency: The Role of N1-Methylpseudouridine in COVID-19 Vaccines.ACS central science · 2021
    Review
16. Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety.Biomedicines · 2021
    Review
17. Current and Future Treatments for Classic Galactosemia.Journal of personalized medicine · 2021
    Review
18. Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency.Proceedings of the National Academy of Sciences of the United States of America · 2019
    Article
19. mRNA rescues neonatal acidemia while mice report no aftereffects.EBioMedicine · 2019
    Article

Corrections and comments

• Erratum for
  Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.2017

Authors and funding

24 authors.

Funding

No grant is acknowledged in the PubMed record.

Abstract

PubMed holds no abstract for this paper.