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Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components.
Kazemian, Pardis; Yu, Si-Yue; Thomson, Sarah B; Birkenshaw, Alexandra; Leavitt, Blair R; Ross, Colin J D.
Afiliação
  • Kazemian P; Department of Medical Genetics, Faculty of Medicine, University of British Columbia, 317-2194 Health Sciences Mall, Vancouver, British Columbia V6T 1Z3, Canada.
  • Yu SY; Centre for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, 938 West 28th Avenue, Vancouver, British Columbia V5Z 4H4, Canada.
  • Thomson SB; Faculty of Pharmaceutical Sciences, University of British Columbia, 2405 Wesbrook Mall, Vancouver, British Columbia V6T 1Z3, Canada.
  • Birkenshaw A; Department of Medical Genetics, Faculty of Medicine, University of British Columbia, 317-2194 Health Sciences Mall, Vancouver, British Columbia V6T 1Z3, Canada.
  • Leavitt BR; Centre for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, 938 West 28th Avenue, Vancouver, British Columbia V5Z 4H4, Canada.
  • Ross CJD; Faculty of Pharmaceutical Sciences, University of British Columbia, 2405 Wesbrook Mall, Vancouver, British Columbia V6T 1Z3, Canada.
Mol Pharm ; 19(6): 1669-1686, 2022 06 06.
Article em En | MEDLINE | ID: mdl-35594500
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Nanopartículas / Edição de Genes Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Nanopartículas / Edição de Genes Idioma: En Ano de publicação: 2022 Tipo de documento: Article