Delivery of gene editing therapeutics

Bhavesh D. Kevadiya, Farhana Islam, Pallavi Deol, Lubaba A. Zaman, Dina A. Mosselhy, Md Ashaduzzaman, Neha Bajwa, Nanda Kishore Routhu, Preet Amol Singh, Shilpa Dawre, Lalitkumar K. Vora, Sumaiya Nahid, Deepali Mathur, Mohammad Ullah Nayan, Ashish Baldi, Ramesh Kothari, Tapan A. Patel, Jitender Madan, Zahra Gounani, Jitender BariwalKenneth S. Hettie, Howard E. Gendelman

Research output: Contribution to journalReview articlepeer-review


For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. Statement of significance: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.

Original languageEnglish (US)
Article number102711
JournalNanomedicine: Nanotechnology, Biology, and Medicine
StatePublished - Nov 2023


  • CRISPR-Cas9
  • Gene editing
  • Lipid nanoparticles
  • Viral vector delivery

ASJC Scopus subject areas

  • Bioengineering
  • Medicine (miscellaneous)
  • Molecular Medicine
  • Biomedical Engineering
  • Materials Science(all)
  • Pharmaceutical Science


Dive into the research topics of 'Delivery of gene editing therapeutics'. Together they form a unique fingerprint.

Cite this