Evidence map›Paper›PMID 40143545›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2025

Clinical development of therapeutic mRNA applications.

Magdalena M Żak, Lior Zangi

Abstract readReview
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

31 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. [Diabetic foot care: full-lifecycle management driven by digital intelligent technologies].Zhongguo xiu fu chong jian wai ke za zhi = Zhongguo xiufu chongjian waike zazhi = Chinese journal of reparative and reconstructive surgery · 2026
    Pooled it
  2. Article
  3. IntegratedMolecular therapy. Nucleic acids · 2026
    Article
  4. Review
  5. Article
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  10. Review
  11. Article
  12. Article
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  14. Review
  15. Article
  16. [Clinical Progress and Prospects of mRNA Tumor Drugs].Sichuan da xue xue bao. Yi xue ban = Journal of Sichuan University. Medical science edition · 2026
    Review
  17. Article
  18. Article
  19. Frontiers in immunology · 2026
    Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

Magdalena M ŻakCardiovascular Research Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA. Electronic address: magdalena.zak@mssm.edu.
Lior ZangiCardiovascular Research Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA. Electronic address: lior.zangi@mssm.edu.

Funding

Project 3 - Role of Proline Metabolism in Regulation of Mammalian Cardiomyocyte ProliferationP01HL160476 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Hesham Sadek · 2022 to 2026
$13.1M
Induction of Cardiomyocyte Proliferation via Transient Expression of Cell Cycle Factors as a Promising Therapy for Heart FailureR01HL147921 · NHLBI · UNIVERSITY OF LOUISVILLE · PI Tamer M A Mohamed, Jonathan Satin · 2020 to 2026
$5.3M
Deciphering the Neonatal Cardiac Regenerative Potential and Regulators in Large AnimalsR01HL149137 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI SADEK, HESHAM, ZANGI, LIOR · 2019 to 2022
$2.5M
Cardiomyocyte-specific modified mRNA of Pkm2 for induction of cardiac regeneration in ischemic heart failureR01HL142768 · NHLBI · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI ZANGI, LIOR · 2018 to 2022
$2.1M
Defining the major signaling mechanism which controls spontaneous cardiomyocyte proliferation in the Neonatal StageR01HL178610 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI Tamer M A Mohamed · 2025 to 2026
$1.5M
An RNA-based approach to intranasal delivery of neuropeptides to the brainR21MH119502 · NIMH · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI HARONY-NICOLAS, HALA, ZANGI, LIOR · 2019 to 2020
$466k
NHLBI NIH HHS P01 HL160476NHLBI NIH HHS R01 HL142768NHLBI NIH HHS R01 HL147921NHLBI NIH HHS R01 HL149137NHLBI NIH HHS R01 HL178610NIMH NIH HHS R21 MH119502
6 · The paper itself

Abstract

mRNA therapeutics are emerging as a transformative approach in modern medicine, providing innovative, highly adaptable solutions for a wide range of diseases, from viral infections to cancer. Since the approval of the first mRNA therapeutic-the coronavirus disease 2019 vaccines in 2021-we have identified more than 70 current clinical trials utilizing mRNA for various diseases. We propose classifying mRNA therapeutics into four main categories: vaccines, protein replacement therapies, antibodies, and mRNA-based cell and gene therapies. Each category can be further divided into subcategories. Vaccines include those targeting viral antigens, bacterial or parasitic antigens, general and individualized cancer antigens, and self-antigens. Protein replacement therapies include maintenance therapeutics designed to treat genetic disorders and interventional therapeutics, where delivering therapeutic proteins could improve patient outcomes, such as vascular endothelial growth factor A for ischemic heart disease or proinflammatory cytokines in cancer. Therapeutic antibodies are based on mRNA sequences encoding the heavy and light chains of clinically relevant antibodies, enabling patient cells to produce them directly, bypassing the costly and complex process of manufacturing protein-ready antibodies. Another category of mRNA-based therapeutics encompasses cell and gene therapies, including CRISPR with mRNA-mediated delivery of Cas9 and the in vivo generation of cells expressing CAR through mRNA. We discuss examples of mRNA therapeutics currently in clinical trials within each category, providing a comprehensive overview of the field's progress and highlighting key advancements as of the end of 2024.

Indexed as

Genetic TherapyRNA, MessengerAnimalsCell- and Tissue-Based TherapyClinical Trials as TopicCOVID-19HumansNeoplasmsSARS-CoV-2RNA, Messengerclinical mRNA therapeuticsmRNA-derived antibodiesmRNA protein replacement therapymRNA therapeuticsmRNA vaccines

Identifiers

PMID40143545
PMCPMC12172183

What OpenQuestion holds

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Registered trials

None linked

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.