Unlocking Genome Editing: Advances and Obstacles in CRISPR/Cas Delivery Technologies

Area of Science:

• Molecular Biology
• Biotechnology
• Genetics

Background:

• CRISPR/Cas9, initially a bacterial immune system, is now a powerful genome-editing tool.
• Its application in mammalian cells enables precise genetic modifications like gene knockouts and regulation.
• CRISPR/Cas9 holds significant promise for treating genetic diseases.

Purpose of the Study:

• To provide a comprehensive review of CRISPR/Cas9 delivery strategies.
• To highlight the limitations of current genome editing technologies.
• To emphasize the need for efficient delivery systems for clinical translation.

Main Methods:

• Review of existing literature on CRISPR/Cas9 delivery.
• Categorization of delivery methods: viral, biomaterials-based, synthetic carriers, and physical techniques.
• Analysis of challenges including off-target effects and immunogenicity.

Main Results:

• CRISPR/Cas9 facilitates diverse genetic manipulations for research and therapeutic development.
• Significant limitations persist, including off-target effects, immunogenicity, and delivery challenges.
• Functional biomaterials and synthetic carriers show promise as effective delivery vehicles.

Conclusions:

• Efficient delivery systems are essential to fully harness CRISPR/Cas9 technology.
• Addressing limitations is key for seamless transition from research to clinical practice.
• Further innovation in delivery methods will accelerate therapeutic applications of genome editing.