The genetic frontier: Transforming therapeutics with CRISPR-Cas9 editing

Linda Chen

doi:10.35841/aabps-15.110.283

Opinion Article - Asian Journal of Biomedical and Pharmaceutical Sciences (2025) Volume 15, Issue 10

The genetic frontier: Transforming therapeutics with CRISPR-Cas9 editing

Linda Chen^*

Department of Pharmacology, National Taiwan University, Taiwan

*Corresponding Author:: Linda Chen
Department of Pharmacology
National Taiwan University, Taiwan
E-mail: Linda.hen@za.tw

Received: 01-Mar-2025, Manuscript No. AABPS-25 -166462; Editor assigned: 03-Mar-2025, Pre QC No. AABPS-25-166462(PQ); Reviewed: 17-Mar-2025, QC No.AABPS-24-166462; Revised: 21-Mar-2025, Manuscript No. AABPS-25-166462(R); Published: 28-Mar-2025, DOI: 10.35841/aabps-15.110.283

Citation: Chen L. The genetic frontier: Transforming therapeutics with CRISPR-Cas9 editing. Asian J Biomed Pharm Sci. 2025;15(110):283

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Introduction

The field of genetic medicine has entered a revolutionary era with the advent of CRISPR-Cas9 gene editing, a powerful tool that allows scientists to modify DNA with unprecedented precision and efficiency. Unlike traditional gene therapy techniques, CRISPR-Cas9 offers a highly specific, programmable method to target and correct genetic mutations at their source. As a result, this genome editing technology holds tremendous potential for curing inherited diseases, developing novel cancer therapies, and reshaping the future of drug discovery and development [1].

CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9) functions as a molecular scissor system, originally derived from the adaptive immune response of bacteria. Its ability to cut DNA at specific sites has rapidly made it the centerpiece of modern genetic engineering [2].

At its core, CRISPR-Cas9 editing operates by utilizing a guide RNA (gRNA) to direct the Cas9 enzyme to a specific sequence of DNA. Once located, the Cas9 enzyme introduces a double-strand break in the target DNA. The cell's natural repair mechanisms—either non-homologous end joining (NHEJ) or homology-directed repair (HDR) then activate, allowing for either gene disruption or the insertion of new genetic material [3].

This precise targeting mechanism allows for manipulation of genetic codes with minimal off-target effects, provided that the guide RNA is carefully designed. The ability to “cut and paste” genes makes CRISPR-Cas9 a transformative force in biomedical research and therapeutic development [4].

CRISPR-Cas9 has vast implications in biomedical research, enabling scientists to create disease models, study gene function, and identify new drug targets. In the pharmaceutical sciences, it is streamlining the drug development pipeline by allowing the generation of genetically modified cell lines and animal models that accurately mimic human diseases [5].

For instance, CRISPR has accelerated the development of personalized cancer therapies by enabling the identification of tumor-specific mutations and creating patient-derived models for drug testing. Additionally, pharmaceutical companies are leveraging CRISPR screens to discover genes responsible for drug resistance, leading to more effective and durable treatments [6].

One of the most promising uses of CRISPR-Cas9 is in curing genetic disorders. Diseases such as sickle cell anemia, beta-thalassemia, and certain forms of retinal degeneration have already shown positive clinical outcomes with CRISPR-based gene editing. By directly correcting pathogenic mutations in hematopoietic stem cells or retinal cells, CRISPR offers a potential one-time cure, as opposed to lifelong symptomatic treatments [7].

In neurological disorders, including Huntington’s disease and amyotrophic lateral sclerosis (ALS), preclinical studies using CRISPR have demonstrated encouraging results. The ability to silence or replace faulty genes in neurons opens the door to therapeutic possibilities that were once considered unattainable [8].

CRISPR-Cas9 is also making waves in the development of next-generation immunotherapies. By modifying T-cells to enhance their cytotoxic capabilities or eliminate inhibitory receptors, researchers are designing more potent CAR-T cell therapies for cancers such as leukemia and lymphoma. Additionally, CRISPR is being used to develop resistance in cells against viruses like HIV by editing out viral DNA from the host genome [9].

In infectious disease research, CRISPR is aiding in the rapid development of vaccines and antiviral drugs. Notably, it has been explored for its potential to edit viral genomes, offering a new dimension in fighting emerging pathogens.

Despite its extraordinary potential, CRISPR-Cas9 editing comes with technical and ethical challenges. One concern is off-target editing, where unintended parts of the genome are modified, potentially leading to harmful mutations. Although advances in high-fidelity Cas9 variants have minimized this risk, comprehensive validation is essential before clinical use.

Ethical debates have intensified following the controversial case of germline editing in human embryos. Regulatory frameworks vary widely between countries, and there is an urgent need for global consensus on the responsible use of CRISPR, especially in reproductive medicine. Issues of informed consent, equity in access, and long-term genetic surveillance also demand careful consideration.

Ongoing research aims to enhance the precision, delivery, and efficiency of CRISPR systems. Innovations such as base editing and prime editing are expanding the editing toolbox, enabling scientists to make even more subtle and controlled genetic modifications. Additionally, non-viral delivery methods, such as lipid nanoparticles, are being explored to improve safety and targeting in clinical applications.

Collaborations between academia, industry, and regulatory agencies are essential to transition CRISPR technologies from bench to bedside. As the understanding of gene function deepens, and editing tools become more refined, CRISPR is poised to become a standard component of therapeutic strategies across a spectrum of diseases [10].

Conclusion

CRISPR-Cas9 gene editing represents a paradigm shift in the treatment of genetic and complex diseases. With its ability to modify DNA at the molecular level, CRISPR is unlocking new possibilities in personalized medicine, immunotherapy, and drug development. While ethical and technical challenges remain, continued innovation and responsible implementation will guide CRISPR from experimental tool to clinical mainstay—reshaping the future of healthcare for generations to come.