Evidence map›Paper›PMID 40777740›Full record

ReviewMolecular therapy. Nucleic acids2025

Recent applications, future perspectives, and limitations of the CRISPR-Cas system.

Sun-Ji Park, Ga Eun Lee, Soo Min Cho, Eui-Hwan Choi

Abstract readReview
In one paragraph

Review 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 17 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed, 1 pooled it
–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

17 citing papers in PubMed, 1 synthesis or guideline pooled it.

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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

4 authors.

Sun-Ji ParkNew Drug Development Center, Daegu-Gyeongbuk Medical Innovation Foundation, Daegu 41061, South Korea.
Ga Eun LeeNew Drug Development Center, Daegu-Gyeongbuk Medical Innovation Foundation, Daegu 41061, South Korea.
Soo Min ChoDepartment of Biotechnology, Korea National University of Transportation, Chungbuk 27909, South Korea.
Eui-Hwan ChoiDepartment of Biotechnology, Korea National University of Transportation, Chungbuk 27909, South Korea.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The CRISPR-Cas system has transformed our ability to edit and modify genomes in eukaryotic cells, offering unmatched precision and broad applicability. By utilizing a programmable RNA protein complex to introduce targeted double-strand breaks, the CRISPR-Cas system enables the correction of pathogenic mutations and the modulation of gene function with unprecedented efficiency. Its broad applicability spans the correction of inherited genetic defects through homology-directed repair to the disruption of deleterious alleles via non-homologous end joining. In this review, we first outline the molecular architecture and mechanistic basis of CRISPR-Cas9 and then consider its latest applications in modeling, drug screening, small-molecule-mediated editing, and treating hereditary, autoimmune, and oncological diseases. Emphasis is placed on the generation of disease-relevant cellular and animal models and on the potential of CRISPR-Cas9-mediated gene therapy to address hitherto intractable disorders. Finally, we discuss current challenges including off-target activity, gene editing efficiency, delivery constraints, and immunogenicity and highlight emerging strategies to overcome these hurdles and broaden the clinical impact of CRISPR-Cas systems.

Indexed as

cell engineeringCRISPR-Cas9DNA repairdrug screeninggene therapygenetic editing efficiencyMT: RNA/DNA Editing

Identifiers

PMID40777740
PMCPMC12329533

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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 Socratic graph.