Role of CRISPR in Developing Personalized Gene Therapy Treatments

DOI: https://doi.org/10.63345/ijrmp.v12.i8.1

Rohit Pillai

Independent Researcher

Kerala, India

Abstract

Recent advances in gene editing have positioned CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) as a pivotal tool in personalized medicine. This manuscript investigates the role of CRISPR in the development of tailored gene therapy treatments. We outline how CRISPR technology has evolved, discuss its mechanism of action, and review preclinical and clinical studies that demonstrate its potential in targeting genetic disorders. Emphasis is placed on studies conducted up to 2021, which highlight successes in correcting gene mutations, as well as the challenges that remain—such as off-target effects, delivery mechanisms, and ethical considerations. The research employs a mixed-methods approach that includes a systematic literature review, a survey among biomedical professionals, and statistical analyses of treatment outcomes from clinical pilot studies. Our findings suggest that while CRISPR-based gene therapy has shown remarkable promise in proof-of-concept trials, broader clinical applications require further refinement. Statistical analysis of early trial data indicates a significant correlation between treatment precision and patient outcomes. The survey results also underscore an optimism tempered by caution among clinicians regarding the long-term implications of CRISPR interventions. The manuscript concludes by discussing future research directions and policy frameworks that could expedite the translation of CRISPR technologies into safe, effective, and personalized therapies.

Keywords

CRISPR, personalized gene therapy, gene editing, clinical trials, genetic disorders, off-target effects

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