Evidence map›Paper›PMID 39967850›Full record

ArticleMolecular therapy. Nucleic acids2025

Use of polyadenosine tail mimetics to enhance mRNA expression from genes associated with haploinsufficiency disorders.

Bahareh Torkzaban, Yining Zhu, Christian Lopez, Jonathan M Alexander, Jingyao Ma, Yongzhi Sun, Katharine R Maschhoff, Wenqian Hu, Michele H Jacob, Dingchang Lin and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–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

6 citing papers in PubMed.

  1. Towards mRNA therapeutics 2.0.Nature reviews. Drug discovery · 2026
    Review
  2. Review
  3. Review
  4. Article
  5. Review
  6. Therapeutic mRNA vaccine applications in oncology.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

13 authors.

Bahareh TorkzabanDepartment of Molecular Biology and Genetics, Johns Hopkins University, Baltimore, MD 21205, USA.
Yining ZhuInstitute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD 21218, USA.
Christian LopezDepartment of Molecular Biology and Genetics, Johns Hopkins University, Baltimore, MD 21205, USA.
Jonathan M AlexanderTufts University School of Medicine, 136 Harrison Avenue, Boston, MA 02111, USA.
Jingyao MaInstitute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD 21218, USA.
Yongzhi SunInstitute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD 21218, USA.
Katharine R MaschhoffDepartment of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, MN 55905, USA.
Wenqian HuDepartment of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, MN 55905, USA.
Michele H JacobTufts University School of Medicine, 136 Harrison Avenue, Boston, MA 02111, USA.
Dingchang LinInstitute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD 21218, USA.
Hai-Quan MaoInstitute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD 21218, USA.
Sophie MartinDepartment of Molecular Biology and Genetics, Johns Hopkins University, Baltimore, MD 21205, USA.
Jeff CollerDepartment of Molecular Biology and Genetics, Johns Hopkins University, Baltimore, MD 21205, USA.

Funding

Development of an oral liver-targeted prime-and-trap malaria vaccineU01AI155313 · NIAID · UNIVERSITY OF WASHINGTON · PI MURPHY, SEAN C · 2021 to 2025
$4.1M
Understanding the relationship between codon optimality and mRNA stabilityR35GM144114 · NIGMS · JOHNS HOPKINS UNIVERSITY · PI Jeffery Coller · 2022 to 2026
$3.0M
Defining the Potential of Gene Therapy to Correct Motor Disabilities of CTNNB1 Syndrome Using in Vivo Mouse and in Vitro Human Cell ModelsR21NS131841 · NINDS · TUFTS UNIVERSITY BOSTON · PI JACOB, MICHELE H. · 2023 to 2023
$454k
NIAID NIH HHS U01 AI155313NIGMS NIH HHS R35 GM144114NINDS NIH HHS R21 NS131841
6 · The paper itself

Abstract

Polyadenosine (poly(A)) tails are nearly ubiquitous in human messenger RNA (mRNA) governing mRNA stability and translation. Crucially, the poly(A) tail regulates cytoplasmic gene expression by undergoing controlled removal upon exposure to the cytoplasm. Upon removal, mRNA ceases protein production and may subsequently be degraded or silenced. We have generated a therapeutic modality that tethers a poly(A) tail mimetic on the 3' end of specifically targeted mRNAs, thereby enhancing their expression beyond their normal utility. This technology, which we term mRNA boosters, lends itself to uses on haploinsufficiency disorders, where reduced gene expression manifests in a disease state. By polyadenylating short RNA sequences antisense to the 3' untranslated region (UTR) of specific mRNAs, we demonstrate that we can selectively and significantly enhance mRNA expression both

Indexed as

antisense oligonucleotidesgene expressionhaploinsufficiency disordersmRNA expressionMT: Oligonucleotides: Therapies and Applicationspolyadenylationpost-transcriptional regulationRNA therapeutics

Identifiers

PMID39967850
PMCPMC11834087

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Registered trials

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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.