CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations
January 2025
in “
Molecules
”
TLDR Non-viral delivery systems and stimuli-responsive nanoformulations can improve CRISPR-Cas9 gene therapy.
The review discusses the challenges and advancements in CRISPR-Cas9 gene therapy, focusing on non-viral delivery systems and stimuli-responsive nanoformulations. It highlights the potential of nonviral vectors like lipid-based nanoparticles, extracellular vesicles, and gold nanoparticles to improve delivery efficiency, stability, and biocompatibility. Stimuli-responsive nanoformulations, which react to pH, redox states, and external triggers, are emphasized for their ability to enhance precision and control in CRISPR-Cas9 delivery, reducing off-target effects. The review underscores the importance of these delivery systems in overcoming current limitations and enhancing the clinical efficacy of CRISPR-Cas9 therapies.