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Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components.

ABSTRACT: Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome editing. Lipid nanoparticles (LNPs) have become an attractive nonviral delivery platform for CRISPR-mediated genome editing due to their low immunogenicity and application flexibility. In this review, we provide a background of CRISPR-mediated gene therapy, as well as LNPs and their applicable characteristics for delivering CRISPR components. We then highlight the challenges of CRISPR delivery, which have driven the significant development of new, safe, and optimized LNP formulations in the past decade. Finally, we discuss considerations for using LNPs to deliver CRISPR and future perspectives on clinical translation of LNP-CRISPR gene editing.

SUBMITTER: Kazemian P

PROVIDER: S-EPMC9176214 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature

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Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components.

Kazemian Pardis P Yu Si-Yue SY Thomson Sarah B SB Birkenshaw Alexandra A Leavitt Blair R BR Ross Colin J D CJD

Molecular pharmaceutics 20220520 6

Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome editing. Lipid nanoparticles (LNPs) have become an attractive nonviral delivery platform for CRISPR-mediated genome editing due to their low immunogenicity and application flexibility. In this review, we provide a bac ...[more]

PMID: 35594500

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