Evidence map›Paper›PMID 40027886›Full record

ArticleMolecular therapy. Nucleic acids2025

Enhanced HDR-mediated correction of heterozygous

John M T Hunt, Alex du Rand, Daniel Verdon, Leah Clemance, Evert Loef, Chloe Malhi, Ben Buttle, David J H F Knapp, Yale S Michaels, Jonathan Garlick and 4 more

Abstract read
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 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. 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

14 authors.

John M T HuntSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Alex du RandSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Daniel VerdonSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Leah ClemanceSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Evert LoefSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Chloe MalhiSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Ben ButtleSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
David J H F KnappInstitut de recherche en immunologie et en cancérologie (IRIC) and Département de Pathologie et Biologie Cellulaire, Université de Montréal, Montréal, QC H3T 1J4, Canada.
Yale S MichaelsPaul Albrechtsen Research Institute CancerCare Manitoba, Winnipeg, MB R2H 2A6, Canada.
Jonathan GarlickSchool of Dental Medicine, Tufts University, Boston, MA 02111, USA.
P Rod DunbarSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Diana PurvisTe Whatu Ora Health New Zealand, Te Toka Tumai, Auckland 1023, New Zealand.
Vaughan FeisstSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.
Hilary SheppardSchool of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene editing facilitated by homology-directed repair (HDR) holds great potential for treating monogenetic disorders such as recessive dystrophic epidermolysis bullosa (RDEB). However, low efficiency and variability between loci must be overcome for its widespread adoption into personalized therapies. To address these challenges, we developed a highly efficient and versatile gene editing strategy for RDEB that incorporates the small molecule inhibitor M3814 to enhance HDR. We focused on three RDEB causative

Indexed as

CRISPR-Cas9dual-nickaseepidermolysis bullosagene therapyhomology-directed repairMT: RNA/DNA EditingOxford Nanopore Technology sequencingskin engineering

Identifiers

PMID40027886
PMCPMC11872078

What OpenQuestion holds

Textmetadata
LicenceCC BY
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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.