Computational tools and resources for CRISPR/Cas genome editing

C Li, W Chu, RA Gill, S Sang, Y Shi, X Hu… - Genomics …, 2023 - academic.oup.com
The past decade has witnessed a rapid evolution in identifying more versatile clustered
regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) …

Evaluating and enhancing target specificity of gene-editing nucleases and deaminases

D Kim, K Luk, SA Wolfe, JS Kim - Annual review of biochemistry, 2019 - annualreviews.org
Programmable nucleases and deaminases, which include zinc-finger nucleases,
transcription activator-like effector nucleases, CRISPR RNA-guided nucleases, and RNA …

Potent and uniform fetal hemoglobin induction via base editing

T Mayuranathan, GA Newby, R Feng, Y Yao… - Nature …, 2023 - nature.com
Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Engineered CRISPR–Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing

BP Kleinstiver, AA Sousa, RT Walton, YE Tak… - Nature …, 2019 - nature.com
Broad use of CRISPR–Cas12a (formerly Cpf1) nucleases has been hindered by the
requirement for an extended TTTV protospacer adjacent motif (PAM). To address this …

Inference of CRISPR edits from Sanger trace data

D Conant, T Hsiau, N Rossi, J Oki, T Maures… - The CRISPR …, 2022 - liebertpub.com
Efficient and precise genome editing requires a fast, quantitative, and inexpensive assay to
assess genotype following editing. Here, we present ICE (Inference of CRISPR Edits), which …

Identifying cell receptors for the nanoparticle protein corona using genome screens

W Ngo, JLY Wu, ZP Lin, Y Zhang, B Bussin… - Nature Chemical …, 2022 - nature.com
Nanotechnology provides platforms to deliver medical agents to specific cells. However, the
nanoparticle's surface becomes covered with serum proteins in the blood after …

Gene correction for SCID-X1 in long-term hematopoietic stem cells

M Pavel-Dinu, V Wiebking, BT Dejene, W Srifa… - Nature …, 2019 - nature.com
Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an
effective therapy for monogenic diseases of the blood and immune system. Here we …

In vivo CRISPR screening reveals nutrient signaling processes underpinning CD8+ T cell fate decisions

H Huang, P Zhou, J Wei, L Long, H Shi, Y Dhungana… - Cell, 2021 - cell.com
How early events in effector T cell (T EFF) subsets tune memory T cell (T MEM) responses
remains incompletely understood. Here, we systematically investigated metabolic factors in …

Precise genome engineering in Drosophila using prime editing

JA Bosch, G Birchak… - Proceedings of the …, 2021 - National Acad Sciences
Precise genome editing is a valuable tool to study gene function in model organisms. Prime
editing, a precise editing system developed in mammalian cells, does not require double …

[HTML][HTML] A genetically engineered primary human natural killer cell platform for cancer immunotherapy

EJ Pomeroy, JT Hunzeker, MG Kluesner, WS Lahr… - Molecular Therapy, 2020 - cell.com
Enhancing natural killer (NK) cell cytotoxicity by blocking inhibitory signaling could lead to
improved NK-based cancer immunotherapy. Thus, we have developed a highly efficient …