Journal
BIOMATERIALS SCIENCE
Volume 10, Issue 23, Pages 6675-6687Publisher
ROYAL SOC CHEMISTRY
DOI: 10.1039/d2bm00806h
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Funding
- National Science Foundation [DGE-1746891]
- NIH NCI [F31CA250367]
- NIH [R01EY031097, P41EB028239, R01CA228133]
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Gene therapies are revolutionizing the treatment of many human diseases, and it is important to consider both the delivery materials and cargos for maximum efficacy and safety. Non-viral delivery mechanisms and CRISPR technology have great potential in immune cell gene editing, but there are challenges that need to be overcome.
Gene therapies are transforming treatment modalities for many human diseases and disorders, including those in ophthalmology, oncology, and nephrology. To maximize the clinical efficacy and safety of these treatments, consideration of both delivery materials and cargos is critical. In consideration of the former, a large effort has been placed on transitioning away from potentially immunoreactive and toxic viral delivery mechanisms towards safer and highly tunable nonviral delivery mechanisms, including polymeric, lipid-based, and inorganic carriers. This change of paradigm does not come without obstacles, as efficient non-viral delivery is challenging, particularly to immune cells, and has yet to see clinical translation breakthroughs for gene editing. This mini-review describes notable examples of biomaterial-based gene delivery to immune cells, with emphasis on recent in vivo successes. In consideration of delivery cargos, clustered regularly interspaced palindromic repeat (CRISPR) technology is reviewed and its great promise in the field of immune cell gene editing is described. This mini-review describes how leading non-viral delivery materials and CRISPR technology can be integrated together to advance its clinical potential for therapeutic gene transfer to immune cells to treat cancer.
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