Browsing Faculty of Arts and Sciences by Keyword "CRISPR/Cas9"
Now showing items 1-5 of 5
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Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles
(Proceedings of the National Academy of Sciences, 2016)A central challenge to the development of protein-based therapeutics is the inefficiency of delivery of protein cargo across the mammalian cell membrane, including escape from endosomes. Here we report that combining ... -
Engineering and evolution of precision genome editing agents
(2022-06-06)Genome editing agents have had a transformative effect on the study of biological systems, the engineering of cells and organisms with novel properties, and even the treatment of genetic diseases in the clinic. This ... -
Expanding the repertoire of methodologies for CRISPR-based genetic manipulation and for hit compound discovery in Mycobacterium abscessus
(2023-11-21)The recent years have seen a rise in infections caused by nontuberculous mycobacteria (NTM), especially by the highly drug-resistant Mycobacterium abscessus. Its large arsenal of intrinsic resistance mechanisms renders ... -
Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies
(Frontiers Media S.A., 2015)Severe combined immunodeficiency (SCID) and other severe non-SCID primary immunodeficiencies (non-SCID PID) can be treated by allogeneic hematopoietic stem cell (HSC) transplantation, but when histocompatibility leukocyte ... -
Quality control, modeling, and visualization of CRISPR screens with MAGeCK-VISPR
(BioMed Central, 2015)High-throughput CRISPR screens have shown great promise in functional genomics. We present MAGeCK-VISPR, a comprehensive quality control (QC), analysis, and visualization workflow for CRISPR screens. MAGeCK-VISPR defines ...