Evidence map›Paper›PMID 42003887›Full record

ArticleMolecular therapy. Nucleic acids2026

CRISPR-Cas9-mediated homology-directed repair rescues the induced bone marrow failure in

Hemavathy Harikrishnan, Mahesh Lamsal, Ka-Kui Chan, Junping Zhang, Jiahe Tian, Kwadwo Fosu, Hong Phuong Nguyen, D Wade Clapp, Elizabeth A Sierra Potchanant, Reuben Kapur and 2 more

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

12 authors.

Hemavathy HarikrishnanHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Mahesh LamsalHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Ka-Kui ChanHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Junping ZhangHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Jiahe TianDepartment of Computer Science, Cornell University, Ithaca, NY, USA.
Kwadwo FosuHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Hong Phuong NguyenHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
D Wade ClappHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Elizabeth A Sierra PotchanantHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Reuben KapurHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Weidong XiaoHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Ngoc Tung TranHerman B Wells Center for pediatric research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.

Funding

Project-005U54DK106846 · NIDDK · INDIANA UNIVERSITY INDIANAPOLIS · PI Reuben Kapur, Karen Elizabeth Pollok · 2015 to 2026
$9.7M
BASIC SCIENCE STUDIES ON GENE THERAPY OF BLOOD DISEASEST32HL007910 · NHLBI · INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS · PI Roland W. Herzog, Reuben Kapur · 1999 to 2026
$7.6M
FANCC mutation correction using homology-independent targeted integration for gene therapy of Fanconi Anemia group CR21HL168669 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI TRAN, NGOC TUNG · 2023 to 2024
$436k
NHLBI NIH HHS R21 HL168669NHLBI NIH HHS T32 HL007910NIDDK NIH HHS U54 DK106846
6 · The paper itself

Abstract

Fanconi anemia (FA) is a rare recessive genetic disorder resulting from mutations in genes in the FA-DNA repair pathway. Among its subtypes, FA complementation group C (

Indexed as

bone marrow failureCRISPR-Cas9FANCCfanconi anemiagene correctiongene therapyhomology-directed repairMT: RNA/DNA editing

Identifiers

PMID42003887
PMCPMC13087461

What OpenQuestion holds

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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.