Evidence map›Paper›PMID 42137269›Full record

ArticleMolecular therapy. Advances2026

Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system.

Monika Chauhan, Audrey L Daugherty, Fatemeh Ellie Khadir, Ozgun F Duzenli, Alexandra Hoffman, Olivia Wan, Jennifer A Tinklenberg, Peter B Kang, George Aslanidi, Christina A Pacak

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Monika ChauhanGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Audrey L DaughertyGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Fatemeh Ellie KhadirGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Ozgun F DuzenliThe Hormel Institute, University of Minnesota, Austin, MN 55912, USA.
Alexandra HoffmanUniversity of Maryland, Baltimore, MD 21201, USA.
Olivia WanGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Jennifer A TinklenbergGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Peter B KangGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
George AslanidiThe Hormel Institute, University of Minnesota, Austin, MN 55912, USA.
Christina A PacakGreg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.

Funding

Gene Therapy for Xeroderma Pigmentosum - Cockayne Syndrome.R61NS135019 · NINDS · UNIVERSITY OF MINNESOTA · PI PACAK, CHRISTINA A. · 2024 to 2025
$849k
FDA HHS R01 FD007483NINDS NIH HHS R61 NS135019
6 · The paper itself

Abstract

Choosing an optimal combination of adeno-associated virus (AAV) capsid and promoter is key to achieving precise, efficient, and safe gene delivery. However, the limited packaging capacity of AAV can make incorporating large transgenes with strong promoters challenging. To address this, we screened several promoter designs derived from portions of the 5' untranslated regions (UTRs) of the human

Indexed as

AAVAAVDJCBAcentral nervous systemEF1αERCC6ERCC8gene therapyhSYNintracerebroventricularintrathecalmini-promoterpromoter

Identifiers

PMID42137269
PMCPMC13148911

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.