International journal of molecular sciences

Tiny Engineered Tools for Gene Therapy: How They Work, Types, and Future Possibilities

Updated

Abstract

Nanotechnology may enhance the precision, efficiency, and safety of genetic modifications in gene therapy.

  • Nanomaterials can address challenges such as inefficient delivery and instability of genetic material in gene therapy.
  • The combination of nanomaterials with CRISPR systems could improve editing specificity and therapeutic effectiveness.
  • Next-generation editing technologies like base editors and prime editing face unique delivery challenges that need to be addressed.
  • Nanocarriers may improve the stability and intracellular delivery of genome-editing systems, potentially leading to more effective treatments for genetic disorders and diseases.
  • Despite advancements, significant challenges including immunotoxicity and regulatory integration remain in translating these technologies to clinical settings.

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Competing interests

0 of 10
authors report competing interests
10 report none
PubMed

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