CRISPR-Cas technologies: Emerging tools from research to clinical application.

Area of Science:

• Biotechnology
• Molecular Biology
• Genetics

Background:

• CRISPR-Cas systems are revolutionary tools in gene therapy.
• Diverse CRISPR-Cas platforms, including SpCas9 variants and other Cas enzymes, have been developed.
• Base editors, prime editors, Cas13, and CRISPRa/i systems expand genome and RNA editing capabilities.

Purpose of the Study:

• To review various CRISPR-based tools and their delivery systems.
• To highlight clinical progress and innovative prospects of CRISPR-Cas technology in gene therapy.
• To discuss the potential of CRISPR-based therapeutics for treating genetic diseases.

Main Methods:

• Review of CRISPR-Cas technologies, including different Cas enzymes and editing systems (base editors, prime editors, Cas13, CRISPRa/i).
• Analysis of various delivery methods for CRISPR-Cas components (electroporation, AAV, lipid nanoparticles).
• Examination of clinical progress and ongoing developments in targeted delivery technologies.

Main Results:

• CRISPR-Cas technology offers versatile gene editing and gene expression regulation.
• Diverse delivery methods are employed to transport CRISPR-Cas components.
• Ongoing advancements focus on improving therapeutic efficiency, safety, and stability.

Conclusions:

• CRISPR-based therapeutics show immense potential for treating genetic diseases through permanent genomic corrections.
• Continued development in targeted delivery is crucial for enhancing therapeutic outcomes.
• CRISPR technology presents innovative prospects for the future of gene therapy.