Evidence map›Paper›PMID 40119516›Full record

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

Recent advances in therapeutic gene-editing technologies.

Dongqi Liu, Di Cao, Renzhi Han

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 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing 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

28 citing papers in PubMed.

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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

3 authors.

Dongqi LiuDepartment of Pediatrics, Department of Molecular and Medical Genetics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Di CaoDepartment of Pediatrics, Department of Molecular and Medical Genetics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Renzhi HanDepartment of Pediatrics, Department of Molecular and Medical Genetics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA. Electronic address: rh11@iu.edu.

Funding

Development of gene editing based therapy for cardiovascular diseasesR01HL159900 · NHLBI · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI CHEN, YUQING EUGENE, HAN, RENZHI · 2021 to 2024
$2.8M
Controllable base editing therapy for DMDR01HL170260 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI Renzhi Han · 2023 to 2026
$2.4M
Targeted editing of ASGR1 for cardiovascular diseasesR01HL169976 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI YUQING Eugene CHEN, Renzhi Han · 2024 to 2026
$2.1M
Base editing of ASGR1 for cardiovascular diseaseR21HL163720 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI HAN, RENZHI · 2023 to 2024
$436k
NHLBI NIH HHS R01 HL159900NHLBI NIH HHS R01 HL169976NHLBI NIH HHS R01 HL170260NHLBI NIH HHS R21 HL163720
6 · The paper itself

Abstract

The advent of gene-editing technologies, particularly CRISPR-based systems, has revolutionized the landscape of biomedical research and gene therapy. Ongoing research in gene editing has led to the rapid iteration of CRISPR technologies, such as base and prime editors, enabling precise nucleotide changes without the need for generating harmful double-strand breaks (DSBs). Furthermore, innovations such as CRISPR fusion systems with DNA recombinases, DNA polymerases, and DNA ligases have expanded the size limitations for edited sequences, opening new avenues for therapeutic development. Beyond the CRISPR system, mobile genetic elements (MGEs) and epigenetic editors are emerging as efficient alternatives for precise large insertions or stable gene manipulation in mammalian cells. These advances collectively set the stage for next-generation gene therapy development. This review highlights recent developments of genetic and epigenetic editing tools and explores preclinical innovations poised to advance the field.

Indexed as

CRISPR-Cas SystemsGene EditingGenetic TherapyAnimalsEpigenesis, GeneticHumansbase editorCRISPRDSBepigenetic editorgene editingoff-target effectsprime editorrecombinasesmall Cas9TALENtransposonsZFN

Identifiers

PMID40119516
PMCPMC12172193

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.