CRISPR/Cas System for Genome Editing: Progress and Prospects as a Therapeutic Tool

Area of Science:

• Molecular Biology
• Biotechnology
• Gene Editing

Background:

• CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) originated from bacterial adaptive immunity against phages.
• The CRISPR/Cas system utilizes a guide RNA and Cas enzyme for targeted DNA modification.
• It has revolutionized biotechnology due to its efficiency, specificity, and reproducibility.

Purpose of the Study:

• To review the CRISPR/Cas system, its history, and classification.
• To discuss the therapeutic applications of CRISPR/Cas in various diseases.
• To explore the role of nonviral vectors in delivering CRISPR/Cas components for therapeutic purposes.

Main Methods:

• Review of CRISPR/Cas system history and mechanisms.
• Analysis of CRISPR/Cas therapeutic potential and limitations.
• Examination of nonviral vector strategies for CRISPR/Cas delivery.

Main Results:

• CRISPR/Cas enables precise gene editing (knockout/knockin) via nonhomologous end joining or homology-directed repair.
• Therapeutic applications are explored for cancer, neurodegenerative diseases, and genetic disorders.
• Nonviral vectors show promise in overcoming delivery challenges like immunogenicity and low transfection.

Conclusions:

• CRISPR/Cas is a powerful tool with significant therapeutic promise.
• Nonviral delivery systems are crucial for advancing CRISPR-based therapies.
• Further research is needed to address limitations for clinical translation.