CRISPR Cas9 revolutionizing genetic engineering and therapeutic applications

Area of Science:

• Biotechnology
• Molecular Biology
• Genetics

Background:

• CRISPR-Cas9, derived from bacterial defense mechanisms, enables targeted DNA manipulation.
• Its precision and adaptability have revolutionized biological research and therapeutic development.

Purpose of the Study:

• To review the transformative impact of CRISPR-Cas9 technology in various scientific and medical fields.
• To explore its applications, potential, and limitations in gene therapy, personalized medicine, and agriculture.

Main Methods:

• CRISPR-Cas9 technology utilizes guide RNA and Cas9 nuclease for targeted DNA cleavage and editing.
• Advanced delivery systems like exosomes and nanoparticles enhance therapeutic efficiency.
• Emerging platforms such as base editing and prime editing offer precise gene regulation without double-strand breaks.

Main Results:

• CRISPR-Cas9 shows significant preclinical success in oncology, particularly in targeting oncogenes and engineering immune cells for CAR-T therapy.
• It holds therapeutic promise for inherited disorders like muscular dystrophy, sickle cell disease, and cystic fibrosis.
• Newer CRISPR-based tools expand therapeutic scope by enabling precise genome and epigenome editing with reduced toxicity.

Conclusions:

• CRISPR-Cas9 is a powerful platform for biotechnology and medicine, with vast potential in treating genetic diseases and cancer.
• Clinical translation is hindered by off-target effects, delivery challenges, ethical concerns, and varying global regulatory landscapes.
• Further research on long-term safety, efficacy, and diverse population applicability is crucial for broader adoption.