CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations

    January 2025 in “ Molecules
    Hyun-Woo Lee, Won‐Yeop Rho, Yoon-Hee Kim, Hyejin Chang, Bong‐Hyun Jun
    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.
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