Harnessing lipid nanoparticles for efficient CRISPR delivery

Abstract

The CRISPR-Cas system has revolutionized the biomedical research field with its simple and flexible genome editing method. In October 2020, Emmanuelle Charpentier and Jennifer A. Doudna were awarded the 2020 Nobel Prize in chemistry in recognition of their outstanding contributions to the discovery of CRISPR-Cas9 genetic scissors, which allow scientists to alter DNA sequences with high precision. Recently, the first phase I clinical trials in cancer patients affirmed the safety and feasibility of ex vivo CRISPR-edited T cells. However, specific and effective CRISPR delivery in vivo remains challenging due to the multiple extracellular and intracellular barriers. Here, we discuss the recent advances in novel lipid nanomaterials for CRISPR delivery and describe relevant examples of potential therapeutics in cancers, genetic disorders, and infectious diseases.