| Literature DB >> 35288219 |
Gyan Prakash1, Ahmed Shokr2, Niels Willemen3, Showkeen Muzamil Bashir4, Su Ryon Shin5, Shabir Hassan6.
Abstract
Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases.Entities:
Keywords: CRISPR-delivery; Gene delivery; Lipid nanoparticles; Microfluidics; Non-viral vectors
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Year: 2022 PMID: 35288219 PMCID: PMC9035142 DOI: 10.1016/j.addr.2022.114197
Source DB: PubMed Journal: Adv Drug Deliv Rev ISSN: 0169-409X Impact factor: 17.873