CRISPR-based gene editing

Overview

CRISPR-based gene editing refers to a family of genome engineering technologies derived from clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins. In biomedical research, these systems are used to alter DNA sequences, disrupt gene function, correct pathogenic variants, or modulate gene expression with high specificity. The platform has become a central tool in functional genomics and translational medicine because it can be adapted for nuclease-mediated cutting, base editing, prime editing, transcriptional activation, and other forms of targeted genetic control.

Biologically, CRISPR-based gene editing is significant because it enables direct interrogation of gene function and disease mechanisms at the level of the genome. Depending on the design, it can be used to create loss-of-function alleles, repair disease-causing mutations, or regulate gene activity without permanently changing the DNA sequence. Its applications span inherited disorders, neurodegeneration, infectious disease, cancer, reproductive medicine, and agriculture, while also raising important ethical questions in contexts such as assisted reproduction, embryology, and preimplantation genetic testing for polygenic risk.

Focus of Latest Publications

Recent publications highlight CRISPR-based gene editing as a versatile platform across both therapeutic development and large-scale functional studies. In a review on bioethics in assisted reproduction and embryology, CRISPR-based gene editing was discussed as one of the future dilemmas alongside in vitro gametogenesis and preimplantation genetic testing for polygenic risk (PGT-P), underscoring its relevance not only as a technical advance but also as a source of emerging ethical and regulatory challenges in reproductive medicine.

In neurodevelopmental disease research, a review of emerging disease-modifying therapies for Angelman syndrome noted that next-generation approaches including CRISPR activation, epigenetic editing, and blood-brain barrier-penetrating delivery systems show preclinical promise. This places CRISPR-based gene editing within a broader strategy of gene regulation rather than only sequence correction, with particular relevance to disorders requiring precise control of neuronal gene expression.

Functional genomics studies continue to rely heavily on CRISPR-based genome editing. A multiplex prime editing framework for identifying drug resistance variants at scale emphasized that CRISPR-based genome editing has revolutionized functional genomics by enabling thousands of perturbations to be assayed concurrently in single experiments. This reflects the use of CRISPR systems as high-throughput discovery tools for mapping genotype–phenotype relationships and identifying variants that influence drug response.

Therapeutic reviews in inherited retinal degenerations described CRISPR as a promising platform for treating inherited retinal diseases, with the cited advances supporting future clinical translation. Although the publication context does not specify individual trial outcomes, it indicates that CRISPR-based strategies are being actively developed for ocular gene correction and disease modification.

In agriculture, CRISPR-based genome editing was presented as part of a translational framework for drought-resilient crop improvement, integrated with metabolomics, pathway engineering, synthetic biology, and artificial intelligence. Here, CRISPR is positioned as a precision breeding tool for modifying traits relevant to climate adaptation.

In neurodegenerative disease research, a review on CRISPR-based correction of apolipoprotein E4 in Alzheimer’s disease described nuclease disruption, base editing, and prime editing as technologies that offer opportunities to modify APOE4 at its genomic source. This connects CRISPR-based gene editing to Alzheimer’s pathology and to broader therapeutic strategies involving antisense oligonucleotide approaches and other macromolecular interventions.

For infectious disease, a review of next-generation gene therapy reported that CRISPR-based genome editing achieved HIV remission in preclinical and early clinical trials. This indicates that CRISPR is being explored not only for ex vivo correction of host cells but also as a potential disease-modifying strategy in persistent viral infection.

In dermatologic genetic disease, a comprehensive review of therapies for epidermolysis bullosa described the clustered regularly interspaced short palindromic repeats/CRISPR-associated protein gene editing landscape for the disorder. This reflects ongoing efforts to use CRISPR-based approaches to address structural gene defects underlying skin fragility syndromes.

CRISPR-based gene editing is also being considered in neuroinflammation and neurodegeneration, where advanced approaches such as CRISPR-based gene editing and RNA therapeutics were described as tools to target molecular mechanisms involved in these disorders. This aligns CRISPR with broader pathway-directed strategies involving oxidative stress, proinflammatory cytokine signaling, mechanistic target of rapamycin kinase, and PRKAA1-related biology.

Finally, a study on spatiotemporally regulated mitochondrial genome editing reported a dual-responsive CRISPR/Cas delivery platform enabling targeted mitochondrial DNA editing for tumor therapy. This expands the scope of CRISPR-based editing beyond the nuclear genome and illustrates the importance of delivery engineering, including nanoparticle systems and near infrared light-responsive control, for achieving spatial and temporal precision.

Key Publications

May A multiplex, prime editing framework for identifying drug resistance variants at scale. (Cell genomics, 2026, PMID 41722572): "CRISPR-based genome editing has revolutionized functional genomics, enabling thousands of perturbations to be concurrently assayed in single experiments."
Apr Metabolomics-guided engineering of drought-resilient crops: Integrating multi-omics and AI for climate-smart agriculture. (Plant science : an international journal of experimental plant biology, 2026, PMID 41662977): "This article emphasizes the integration of metabolomics with cutting-edge technologies, CRISPR-based genome editing, pathway engineering, synthetic biology, and artificial intelligence, to establish a translational framework for drought-resilient crop improvement."
Apr Spatiotemporally regulated mitochondrial genome editing via enzyme and NIR-activated CRISPR/Cas9 nanoplatform. (Chemical science, 2026, PMID 41584446): "Herein, we report a dual-responsive CRISPR/Cas delivery platform (UCRP-TPP) that enables spatiotemporally regulated mtDNA editing for targeted tumor therapy."
Apr Emerging disease-modifying therapies for Angelman syndrome: A comprehensive review for pediatric neurologists. (Brain & development, 2026, PMID 41864145): "Next-generation approaches including CRISPR activation, epigenetic editing, and blood-brain barrier-penetrating delivery systems show preclinical promise."
Apr Neuroinflammation, Autophagy, and Neurodegeneration: Mechanisms and Therapeutic Insights. (CNS & neurological disorders drug targets, 2026, PMID 41918200): "Advanced approaches such as CRISPR-based gene editing and RNA therapeutics provide tools to target molecular mechanisms involved in these neurodegenerative disorders."
Apr Next-generation gene therapy for infectious disease: Advances, challenges, and future directions. (Journal of infection and public health, 2026, PMID 41671593): "Findings show CRISPR-based genome editing achieved HIV remission in preclinical and early clinical trials."
Apr CRISPR as a therapeutic tool for inherited retinal degenerations: Advances, challenges, and future directions. (Molecular aspects of medicine, 2026, PMID 41702027): "Together, these advances underscore the transformative potential of CRISPR technologies in treating IRDs and lay the foundation for future clinical translation."
Apr Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies. (Drugs, 2026, PMID 41731282): "Here, we comprehensively describe the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) gene editing landscape for EB."
Apr CRISPR-based correction of apolipoprotein E4 in Alzheimer's disease: Therapeutic strategies and macromolecular delivery innovations. (International journal of biological macromolecules, 2026, PMID 41812941): "CRISPR-based genome editing technologies, including nuclease disruption, base editing, and prime editing, offer unprecedented opportunities to directly modify APOE4 at its genomic source."
Apr Bioethics in assisted reproduction and embryology. (Systems biology in reproductive medicine, 2026, PMID 41581067): "...future dilemmas posed by CRISPR-based gene editing, IVG, and preimplantation genetic testing for polygenic risk (PGT-P)."