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Literature DB >> 35594500

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

Pardis Kazemian^1,2, Si-Yue Yu³, Sarah B Thomson^1,2, Alexandra Birkenshaw³, Blair R Leavitt^1,2, Colin J D Ross³.

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.

Entities: Chemical

Keywords: CRISPR/Cas9; gene therapy; genome editing; lipid nanoparticles; nanomedicine

Mesh：

Substances：

Year: 2022 PMID： 35594500 PMCID： PMC9176214 DOI： 10.1021/acs.molpharmaceut.1c00916

Source DB: PubMed Journal: Mol Pharm ISSN： 1543-8384 Impact factor: 5.364

94 in total

1. Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA.

Authors: Katalin Karikó; Michael Buckstein; Houping Ni; Drew Weissman
Journal: Immunity Date: 2005-08 Impact factor: 31.745

2. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability.

Authors: Katalin Karikó; Hiromi Muramatsu; Frank A Welsh; János Ludwig; Hiroki Kato; Shizuo Akira; Drew Weissman
Journal: Mol Ther Date: 2008-09-16 Impact factor: 11.454

3. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

Authors: Akin Akinc; William Querbes; Soma De; June Qin; Maria Frank-Kamenetsky; K Narayanannair Jayaprakash; Muthusamy Jayaraman; Kallanthottathil G Rajeev; William L Cantley; J Robert Dorkin; James S Butler; Liuliang Qin; Timothy Racie; Andrew Sprague; Eugenio Fava; Anja Zeigerer; Michael J Hope; Marino Zerial; Dinah W Y Sah; Kevin Fitzgerald; Mark A Tracy; Muthiah Manoharan; Victor Koteliansky; Antonin de Fougerolles; Martin A Maier
Journal: Mol Ther Date: 2010-05-11 Impact factor: 11.454

4. The role of apolipoprotein E in the elimination of liposomes from blood by hepatocytes in the mouse.

Authors: Xuedong Yan; Folkert Kuipers; Louis M Havekes; Rick Havinga; Bert Dontje; Klaas Poelstra; Gerrit L Scherphof; Jan A A M Kamps
Journal: Biochem Biophys Res Commun Date: 2005-03-04 Impact factor: 3.575

5. Nanoparticle depots for controlled and sustained gene delivery.

Authors: Zhongyu Li; William Ho; Xin Bai; Fengqiao Li; Yen-Jui Chen; Xue-Qing Zhang; Xiaoyang Xu
Journal: J Control Release Date: 2020-03-16 Impact factor: 9.776

Review 6. The Biomolecular Corona of Lipid Nanoparticles for Gene Therapy.

Authors: Valentina Francia; Raymond M Schiffelers; Pieter R Cullis; Dominik Witzigmann
Journal: Bioconjug Chem Date: 2020-08-31 Impact factor: 4.774

7. In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA.

Authors: Lukas Villiger; Tanja Rothgangl; Dominik Witzigmann; Rurika Oka; Paulo J C Lin; Weihong Qi; Sharan Janjuha; Christian Berk; Femke Ringnalda; Mitchell B Beattie; Markus Stoffel; Beat Thöny; Jonathan Hall; Hubert Rehrauer; Ruben van Boxtel; Ying K Tam; Gerald Schwank
Journal: Nat Biomed Eng Date: 2021-01-25 Impact factor: 25.671

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

1. Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA.

2. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability.

3. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

4. The role of apolipoprotein E in the elimination of liposomes from blood by hepatocytes in the mouse.

5. Nanoparticle depots for controlled and sustained gene delivery.

Review 6. The Biomolecular Corona of Lipid Nanoparticles for Gene Therapy.

7. In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA.

Review 8. Engineering precision nanoparticles for drug delivery.

Review 9. mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability.

Review 10. Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.

1. The promise of gene editing: so close and yet so perilously far.

Review 2. Nanocarriers: A novel strategy for the delivery of CRISPR/Cas systems.