Nanoparticle-enhanced CRISPR delivery: paving the path for <i>in vivo</i> tumor gene editing


Khizar M., Aminpoor H., Zaib M., Ali Q., Karimi H.

ANNALS OF MEDICINE AND SURGERY, cilt.88, sa.1, ss.1054-1055, 2026 (ESCI) identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 88 Sayı: 1
  • Basım Tarihi: 2026
  • Doi Numarası: 10.1097/ms9.0000000000004414
  • Dergi Adı: ANNALS OF MEDICINE AND SURGERY
  • Derginin Tarandığı İndeksler: Emerging Sources Citation Index (ESCI)
  • Sayfa Sayıları: ss.1054-1055
  • Bezmiâlem Vakıf Üniversitesi Adresli: Evet

Özet

Nanoparticle-based delivery systems are redefining how CRISPR/Cas technology can be used in cancer treatment. By encapsulating CRISPR components within lipid, polymeric, or inorganic nanoparticles, researchers have improved their stability, circulation time, and tumor-targeting precision. The NTLA-2001 trial demonstrated the first successful use of lipid nanoparticles for in vivo CRISPR delivery in humans, paving the way for potential applications in oncology. Preclinical studies have shown promising results, with efficient gene knockout and tumor suppression across multiple models. Despite these advances, barriers remain, including limited delivery to solid tumors, potential off-target effects, and inconsistent nanoparticle formulations. Global research efforts spanning the United States, China, Europe, and India are now focused on refining delivery platforms and standardizing protocols. This letter highlights current progress, ongoing challenges, and the need for transparent, globally coordinated development. Nanoparticle-enhanced CRISPR delivery has the potential to bring genetic precision therapy from the laboratory to the clinic, offering a new avenue for durable and accessible cancer care.