Current applications and future perspective of CRISPR/Cas9 gene editing in cancer

SW Wang, C Gao, YM Zheng, L Yi, JC Lu, XY Huang… - Molecular cancer, 2022 - Springer
Clustered regularly interspaced short palindromic repeats (CRISPR) system provides
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …

Lipids and lipid derivatives for RNA delivery

Y Zhang, C Sun, C Wang, KE Jankovic… - Chemical reviews, 2021 - ACS Publications
RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models

RG Lee, AM Mazzola, MC Braun, C Platt, SB Vafai… - Circulation, 2023 - ahajournals.org
Background: VERVE-101 is an investigational in vivo CRISPR base-editing medicine
designed to alter a single DNA base in the PCSK9 gene, permanently turn off hepatic …

Adenine transversion editors enable precise, efficient A• T-to-C• G base editing in mammalian cells and embryos

L Chen, M Hong, C Luan, H Gao, G Ru, X Guo… - Nature …, 2024 - nature.com
Base editors have substantial promise in basic research and as therapeutic agents for the
correction of pathogenic mutations. The development of adenine transversion editors has …