Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 3 complete abstracts (616 words), 9 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
Genetic engineering or molecular biology techniques that involve dna repair mechanisms for incorporating site-specific modifications into a cell's genome.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Editing, Gene; Genome Editing; Editing, Genome; Base Editing; Editing, Base.
Where it fits in the MeSH classification
- Broader topics
- Genetic Engineering
- See also
- Mutagenesis, Site-Directed; CRISPR-Cas Systems; CRISPR-Associated Protein 9
Related concepts
Research landscape
PubMed indexes 18,185 records tagged with the MeSH heading “Gene Editing” (counts retrieved 2026-10-29). 12,773 of them (70.2%) were published from 2020 onward, and the busiest period so far is 2020 onward with 12,773 records.
Reviews account for 4,903 records (27.0%) and meta-analyses for 18. Randomized controlled trials number 2 (0.0%) as a share of all records on the topic. Randomized trials make up only a small share of this literature, so much of what is known comes from laboratory, observational or descriptive work.
Show the data
| Period | Records |
|---|---|
| through 1999 | 0 |
| 2000-2009 | 0 |
| 2010-2019 | 5,599 |
| 2020 onward | 12,773 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 4,903 | 27.0% |
| Meta-analyses | 18 | 0.1% |
| Randomized controlled trials | 2 | 0.0% |
Method: PubMed query "Gene Editing"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-29.
Reading the evidence in genetics and genomics
Genetic findings vary widely in strength. A variant linked to a condition in one study may not replicate in other ancestries, and most common variants have small effects that are meaningful only in aggregate. Studies of rare, high-impact variants can support strong causal claims about a gene, while association studies describe statistical links that need functional and independent replication. Check the sample size, the ancestry of participants, whether results were replicated, and whether a laboratory or clinical outcome was actually measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| In vivo site-specific engineering to reprogram T cells | 2026 | Research article Design not recorded in structured metadata | 33 | cc by PMID 41851456 · DOI · PMC13083257 |
| High-efficiency, transgene-free plant genome editing by viral delivery of an engineered TnpB | 2026 | Research article Design not recorded in structured metadata | 14 | cc by PMID 41720886 · DOI · PMC13013028 |
| Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders | 2026 | Research article Design not recorded in structured metadata | 12 | cc by PMID 41951737 · DOI · PMC13253345 |
In vivo site-specific engineering to reprogram T cells [1]
Abstract reading summary — source excerpts
- Opening excerpt
- Engineered T cells, reprogrammed to express chimeric antigen receptors (CAR) or T cell receptors (TCR), have transformed cancer treatment and are being explored as therapeutics for autoimmune and infectious diseases.
- Closing excerpt
- These findings offer a pathway to more efficient, precise and widely accessible T cell therapies.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (200 words)
Engineered T cells, reprogrammed to express chimeric antigen receptors (CAR) or T cell receptors (TCR), have transformed cancer treatment and are being explored as therapeutics for autoimmune and infectious diseases. Enhancing T cell function through genome editing, either by disrupting endogenous genes or precisely inserting DNA payloads, has shown considerable promise1. However, the ex vivo manufacturing process is lengthy and costly, limiting accessibility of these therapies. In vivo generation of CAR T cells could overcome these barriers, but current methods rely either on transient expression with limited durability, or on random integration of DNA payloads that lack specificity. Here we demonstrate that stable and cell-specific transgene expression can be achieved through in vivo site-specific integration of large DNA payloads. We developed a two-vector system to deliver CRISPR-Cas9 ribonucleoproteins and a DNA donor template, using enveloped delivery vehicles and adeno-associated viruses, respectively. We optimized both vectors for T cell-specific delivery and gene-targeting efficiency. By integrating a CAR transgene into a T cell-specific locus, we generate therapeutic levels of CAR T cells in vivo in humanized mouse models of B cell aplasia, and haematological and solid malignancies. These findings offer a pathway to more efficient, precise and widely accessible T cell therapies.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41851456 · DOI · PMC13083257
High-efficiency, transgene-free plant genome editing by viral delivery of an engineered TnpB [2]
Abstract reading summary — source excerpts
- Opening excerpt
- Genome editing has revolutionized plant biology research1, yet the efficient delivery of editing reagents remains a challenge.
- Closing excerpt
- The design principles outlined here could promote wider use of eTnpBc for efficient, transformation- and transgene-free plant genome editing.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (204 words)
Genome editing has revolutionized plant biology research1, yet the efficient delivery of editing reagents remains a challenge. Current methods are labour intensive, involving lengthy tissue culture and complex transformation and regeneration steps. Viral delivery can mitigate these issues2 but CRISPR-Cas nucleases exceed viral cargo limits, restricting guide RNA (gRNA) delivery into Cas9-expressing transgenic plants2-11. This requires generating an initial Cas9 transgenic line. Furthermore, gRNAs delivered by plant viral vectors can induce somatic edits, although only a few produce heritable edits3-7,9-12. Some engineered plant negative-strand rhabdoviruses can deliver both Cas9 and gRNA, but they face other challenges, including the need for tissue regeneration or pruning infected plants, and some rhabdoviruses can be delivered only through vector transmission13-16. Recently, smaller editors such as TnpBs were discovered, but they are significantly less active than Cas917-19. Here we optimized a tobacco rattle virus-based system to deliver recently engineered, highly active ISDra2 TnpB variants. The eTnpBc variant enables effective somatic editing in systemic leaves and achieves up to 90% editing efficiency at target loci. In addition, up to 89% of offspring exhibit a mutant phenotype, with editing efficiencies reaching 100%. The design principles outlined here could promote wider use of eTnpBc for efficient, transformation- and transgene-free plant genome editing.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41720886 · DOI · PMC13013028
Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Recently, de novo variants in an 18-nucleotide region in the centre of RNU4-2 were shown to cause ReNU syndrome, a syndromic neurodevelopmental disorder that is predicted to affect tens of thousands of individuals worldwide1,2.
- Closing excerpt
- Together, this work defines the landscape of variant function across RNU4-2, providing critical insights for both diagnosis and therapeutic development.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (212 words)
Recently, de novo variants in an 18-nucleotide region in the centre of RNU4-2 were shown to cause ReNU syndrome, a syndromic neurodevelopmental disorder that is predicted to affect tens of thousands of individuals worldwide1,2. RNU4-2 is a non-protein-coding gene that is transcribed into the U4 small nuclear RNA component of the major spliceosome3. ReNU syndrome variants disrupt spliceosome function and alter 5' splice site selection1,4. Here we performed saturation genome editing (SGE) of RNU4-2 to identify the functional and clinical impact of variants across the entire gene. The resulting SGE function scores, derived from variants' effects on cell fitness, discriminate ReNU syndrome variants from those observed in the population and markedly outperform in silico variant effect prediction. Using these data, we redefine the ReNU syndrome critical region at single-nucleotide resolution, resolve variant pathogenicity for variants of uncertain significance and show that SGE function scores delineate variants by phenotypic severity and the extent of observed splicing disruption. Furthermore, we identify variants affecting function in regions of RNU4-2 that are critical for interactions with other spliceosome components. We show that these variants cause a new recessive neurodevelopmental disorder that is distinct from ReNU syndrome. Together, this work defines the landscape of variant function across RNU4-2, providing critical insights for both diagnosis and therapeutic development.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41951737 · DOI · PMC13253345
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article An overview and metanalysis of machine and deep learning-based CRISPR gRNA design tools [4]
- Research article The hidden risks of CRISPR/Cas: structural variations and genome integrity [5]
- Research article Gene therapy and genome editing for lipoprotein disorders [6]
- Research article Revolutionizing CRISPR technology with artificial intelligence [7]
- Research article Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta-analysis of preclinical studies [8]
- Research article Harnessing artificial intelligence to advance CRISPR-based genome editing technologies [9]
References
- Nyberg WA, Bernard PL, Ngo W, Wang CH, Ark J, Rothrock A, et al.. In vivo site-specific engineering to reprogram T cells. . 2026. PMID 41851456 · DOI · PMC13083257
- Nagalakshmi U, Rodriguez JE, Nguyen T, Weissman RF, Thornton BW, Terrace CI, et al.. High-efficiency, transgene-free plant genome editing by viral delivery of an engineered TnpB. . 2026. PMID 41720886 · DOI · PMC13013028
- De Jonghe J, Kim HC, Adedeji A, Leitão E, Dawes R, Kajba CM, et al.. Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders. . 2026. PMID 41951737 · DOI · PMC13253345
- Wang J, Zhang X, Cheng L, Luo Y. An overview and metanalysis of machine and deep learning-based CRISPR gRNA design tools. RNA Biol. 2020. PMID 31533522 · DOI · PMC6948960
- Aussel C, Cathomen T, Fuster-García C. The hidden risks of CRISPR/Cas: structural variations and genome integrity. Nat Commun. 2025. PMID 40764301 · DOI · PMC12325979
- Gurevitz C, Bajaj A, Khera AV, Do R, Schunkert H, Musunuru K, et al.. Gene therapy and genome editing for lipoprotein disorders. Eur Heart J. 2025. PMID 40600248 · DOI · PMC12448413
- Kim MG, Go MJ, Kang SH, Jeong SH, Lim K. Revolutionizing CRISPR technology with artificial intelligence. Exp Mol Med. 2025. PMID 40745000 · DOI · PMC12322281
- Maganti HB, Bailey AJM, Kirkham AM, Shorr R, Pineault N, Allan DS. Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta-analysis of preclinical studies. Stem Cells Transl Med. 2021. PMID 33666363 · DOI · PMC8235122
- Thomson T, Li G, Strilchuk A, Cui H, Wang B, Li B. Harnessing artificial intelligence to advance CRISPR-based genome editing technologies. Nat Rev Genet. 2026. PMID 41254174 · DOI
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-29.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.