Overcoming the delivery problem for therapeutic genome editing: Current status and perspective of non-viral methods

TV Mashel, YV Tarakanchikova, AR Muslimov… - Biomaterials, 2020 - Elsevier
Besides its broad application in research and biotechnology, genome editing (GE) has great
potential for clinical gene therapy, but delivery of GE tools remains a bottleneck. Whereas …

Gene editing and its applications in biomedicine

G Li, X Li, S Zhuang, L Wang, Y Zhu, Y Chen… - Science China Life …, 2022 - Springer
The steady progress in genome editing, especially genome editing based on the use of
clustered regularly interspaced short palindromic repeats (CRISPR) and programmable …

Generation and comparative analysis of an Itga8-CreERT2 mouse with preferential activity in vascular smooth muscle cells

G Warthi, JL Faulkner, J Doja, AR Ghanam… - Nature cardiovascular …, 2022 - nature.com
All current smooth muscle cell (SMC) Cre mice similarly recombine floxed alleles in vascular
and visceral SMCs. Here we present an Itga8-CreER T2 knock-in mouse and compare its …

Mouse gene-targeting strategies for maximum ease and versatility

VE Papaioannou, RR Behringer - Cold Spring Harbor …, 2024 - cshprotocols.cshlp.org
Well-planned strategies are an essential prerequisite for any mutational analysis involving
gene targeting. Consideration of the advantages or disadvantages of different methods will …

[HTML][HTML] “Genetic scissors” CRISPR/Cas9 genome editing cutting-edge biocarrier technology for bone and cartilage repair

C Li, Y Du, T Zhang, H Wang, Z Hou, Y Zhang, W Cui… - Bioactive Materials, 2023 - Elsevier
CRISPR/Cas9 is a revolutionary genome editing technology with the tremendous
advantages such as precisely targeting/shearing ability, low cost and convenient operation …

Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integrase

BE Low, V Hosur, S Lesbirel, MV Wiles - Scientific Reports, 2022 - nature.com
The development of mouse models of human disease and synthetic biology research by
targeted transgenesis of large DNA constructs represent a significant genetic engineering …

Rare SH2B3 coding variants in lupus patients impair B cell tolerance and predispose to autoimmunity

Y Zhang, R Morris, GJ Brown, AMD Lorenzo… - Journal of Experimental …, 2024 - rupress.org
Systemic lupus erythematosus (SLE) is a heterogeneous autoimmune disease with a clear
genetic component. While most SLE patients carry rare gene variants in lupus risk genes …

DECTIN-1: A modifier protein in CTLA-4 haploinsufficiency

C Turnbull, J Bones, M Stanley, A Medhavy… - Science …, 2023 - science.org
Autosomal dominant loss-of-function (LoF) variants in cytotoxic T-lymphocyte associated
protein 4 (CTLA4) cause immune dysregulation with autoimmunity, immunodeficiency and …

LRRC8A is essential for hypotonicity-, but not for DAMP-induced NLRP3 inflammasome activation

JP Green, T Swanton, LV Morris, LY El-Sharkawy… - Elife, 2020 - elifesciences.org
The NLRP3 inflammasome is a multi-molecular protein complex that converts inactive
cytokine precursors into active forms of IL-1β and IL-18. The NLRP3 inflammasome is …

A most formidable arsenal: genetic technologies for building a better mouse

JF Clark, CJ Dinsmore, P Soriano - Genes & Development, 2020 - genesdev.cshlp.org
The mouse is one of the most widely used model organisms for genetic study. The tools
available to alter the mouse genome have developed over the preceding decades from …