Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing.

Area of Science:

• Biotechnology and Biomedical Engineering
• Molecular Biology and Genetics
• Nanomedicine

Background:

• Genome editing, particularly CRISPR/Cas technology, holds significant potential for treating intractable diseases.
• Current clinical applications of CRISPR/Cas are hindered by challenges in delivering the system and single-guide RNAs (sgRNAs) into target cells.

Purpose of the Study:

• To review recent advancements in nonviral, nanovesicle-based delivery systems for genome-editing technologies.
• To explore strategies for enhancing CRISPR/Cas and sgRNA delivery using nanovesicles.
• To identify future research directions for nanovesicle-mediated genome editing.

Main Methods:

• Investigation of various nonviral nanovesicle systems, including lipid-, polymer-, peptide-, and extracellular vesicle-based carriers.
• Design of nanovesicles for improved endosomal escape and targeted delivery.
• Utilization of stimuli-responsive features (light, pH, environmental cues) to trigger payload release.

Main Results:

• Nanovesicles offer a promising alternative to viral vectors for delivering CRISPR/Cas and sgRNA.
• Engineered nanovesicles demonstrate enhanced cellular uptake and payload delivery efficiency.
• Stimuli-responsive nanovesicles provide spatiotemporal control over genome editing processes.

Conclusions:

• Nonviral nanovesicle-based delivery systems are crucial for realizing the therapeutic potential of genome editing.
• Continued research into nanovesicle design and stimuli-responsive mechanisms will accelerate clinical translation.
• Optimized nanovesicle delivery is key to overcoming current barriers in genome-editing therapies.