CRISPR/Cas9: Principle, Applications, and Delivery through Extracellular Vesicles

Area of Science:

• Molecular Biology
• Biotechnology
• Genetics

Background:

• CRISPR/Cas9 technology revolutionized eukaryotic gene editing for research and therapeutics.
• Clinical applications are hindered by inefficient and unsafe delivery of CRISPR/Cas9 components in vivo.
• Challenges include off-target effects, immune responses, toxicity, and rapid degradation of delivery vehicles.

Purpose of the Study:

• To review advancements in CRISPR/Cas9 technology.
• To explore the use of extracellular vesicles for CRISPR/Cas9 delivery.
• To summarize the current state of using exosomes and microvesicles for intracellular transport of CRISPR/Cas9.

Main Methods:

• Review of CRISPR/Cas9 system advancements and applications.
• Analysis of extracellular vesicles (exosomes, microvesicles) as delivery systems.
• Summary of current research on extracellular vesicle-mediated CRISPR/Cas9 delivery.

Main Results:

• CRISPR/Cas9 technology has evolved beyond simple gene disruption to versatile gene modification tools.
• Extracellular vesicles show potential to overcome delivery challenges associated with CRISPR/Cas9.
• Exosomes and microvesicles are being investigated for efficient intracellular transport of CRISPR/Cas9.

Conclusions:

• Efficient and safe delivery of CRISPR/Cas9 remains a critical bottleneck for therapeutic applications.
• Extracellular vesicles represent a promising strategy to enhance in vivo delivery of CRISPR/Cas9 components.
• Further research into extracellular vesicle-mediated delivery could unlock the full therapeutic potential of CRISPR/Cas9.