Advanced International Journal for Research

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Volume 7 Issue 4
July-August 2026

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Nanoparticle-Mediated CRISPR-Cas9 Delivery: Current Strategies, Pharmacological Challenges and Therapeutic Applications in Genetic Disorders

Author(s) Mr. Shouvik Mondal, Mr. Kriti Kumari
Country India
Abstract The CRISPR-Cas9 genome-editing system has emerged as a promising technology for the precise correction of disease-causing mutations and has shown significant potential in treating various genetic disorders. However, the successful clinical application of CRISPR-Cas9 largely depends on the development of safe and efficient delivery systems. Among the available approaches, nanoparticle-mediated delivery has gained considerable attention for its low immunogenicity, high biocompatibility, improved targeting, and capacity to deliver diverse CRISPR cargoes. This review summarises the current strategies of nanoparticle-mediated CRISPR-Cas9 delivery, including lipid, polymeric, hybrid, inorganic, and biomimetic nanoparticles. It also discusses major pharmacological challenges such as off-target effects, biodistribution, endosomal escape, immunogenicity, toxicity, and regulatory considerations. Furthermore, recent therapeutic applications in genetic disorders, including sickle cell disease, β-thalassemia, Duchenne muscular dystrophy, cystic fibrosis, hereditary transthyretin amyloidosis, and cancer, are highlighted. Finally, future perspectives on advanced nanocarrier design and clinical translation are discussed. Nanoparticle-mediated CRISPR-Cas9 delivery holds great promise for advancing precision medicine and developing effective therapies for genetic disorders.
Keywords CRISPR-Cas9; Nanoparticles; Genome editing; Genetic disorders; Gene therapy; Drug delivery
Field Chemistry > Pharmacy
Published In Volume 7, Issue 4, July-August 2026
Published On 2026-08-01
DOI https://doi.org/10.63363/aijfr.2026.v07i04.7111
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