Results 31 to 40 of about 254,775 (306)

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

New England Journal of Medicine, 2020
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in
H. Frangoul, D. Altshuler, M. Cappellini, Yi-Sha Chen, J. Domm, Brenda K. Eustace, J. Foell, J. de la Fuente, S. Grupp, R. Handgretinger, Tony Ho, A. Kattamis, A. Kernytsky, J. Lekstrom-Himes, Amanda M Li, F. Locatelli, M. Mapara, M. de Montalembert, D. Rondelli, Akshay Sharma, S. Sheth, S. Soni, M. Steinberg, D. Wall, Angela Yen, S. Corbacioglu   +25 more
semanticscholar   +1 more source

Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing

Cell Reports, 2019
Summary: CRISPR-Cas9 systems provide powerful tools for genome editing. However, optimal employment of this technology will require control of Cas9 activity so that the timing, tissue specificity, and accuracy of editing may be precisely modulated.
Bianca Garcia, Jooyoung Lee, Alireza Edraki, Yurima Hidalgo-Reyes, Steven Erwood, Aamir Mir, Chantel N. Trost, Uri Seroussi, Sabrina Y. Stanley, Ronald D. Cohn, Julie M. Claycomb, Erik J. Sontheimer, Karen L. Maxwell, Alan R. Davidson   +13 more
doaj   +1 more source

MAGeCK enables robust identification of essential genes from genome-scale CRISPR/Cas9 knockout screens

Genome Biology, 2014
We propose the Model-based Analysis of Genome-wide CRISPR/Cas9 Knockout (MAGeCK) method for prioritizing single-guide RNAs, genes and pathways in genome-scale CRISPR/Cas9 knockout screens.
Wei Li, Han Xu, T. Xiao, Lê Công, Lê Công, M. Love, Feng Zhang, Feng Zhang, R. Irizarry, Jun S. Liu, Myles Brown, Myles Brown, X. Liu   +12 more
semanticscholar   +1 more source

The MyLo CRISPR-Cas9 Toolkit: A Markerless Yeast Localization and Overexpression CRISPR-Cas9 Toolkit [PDF]

G3 Genes|Genomes|Genetics, 2021
ABSTRACT The genetic tractability of the yeast Saccharomyces cerevisiae has made it a key model organism for basic research and a target for metabolic engineering. To streamline the introduction of tagged genes and compartmental markers with powerful CRISPR-Cas9-based genome editing tools we constructed a Markerless Yeast Localization ...
Björn D M Bean, Malcolm Whiteway, Vincent J J Martin   +2 more
openaire   +2 more sources

Genetic screens in human cells using the CRISPR/Cas9 system

Science, 2013
Improving Whole-Genome Screens Improved methods are needed for the knockout of individual genes in genome-scale functional screens. Wang et al. (p. 80, published online 12 December) and Shalem et al. (p.
Tim Wang, Jenny J. Wei, D. Sabatini, E. Lander   +3 more
semanticscholar   +1 more source

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

Nature, 2014
Systematic interrogation of gene function requires the ability to perturb gene expression in a robust and generalizable manner. Here we describe structure-guided engineering of a CRISPR-Cas9 complex to mediate efficient transcriptional activation at ...
S. Konermann, Mark D. Brigham, Alexandro E. Trevino, J. Joung, O. Abudayyeh, Clea Bárcena, P. Hsu, Naomi Habib, J. Gootenberg, H. Nishimasu, O. Nureki, Feng Zhang   +11 more
semanticscholar   +1 more source

Unconstrained Genome Targeting with near-PAMless Engineered CRISPR-Cas9 Variants

Science, 2020
A PAMless base editor CRISPR-Cas DNA base editing typically requires a specific motif for targeting known as a protospacer-adjacent motif (PAM). This requirement limits the sequences within a genome that can be targeted. Walton et al. engineered specific
R. Walton, Kathleen A. Christie, Madelynn N. Whittaker, B. Kleinstiver   +3 more
semanticscholar   +1 more source

Using local chromatin structure to improve CRISPR/Cas9 efficiency in zebrafish. [PDF]

PLoS ONE, 2017
Although the CRISPR/Cas9 has been successfully applied in zebrafish, considerable variations in efficiency have been observed for different gRNAs. The workload and cost of zebrafish mutant screening is largely dependent on the mutation rate of injected ...
Yunru Chen, Shiyang Zeng, Ruikun Hu, Xiangxiu Wang, Weilai Huang, Jiangfang Liu, Luying Wang, Guifen Liu, Ying Cao, Yong Zhang   +9 more
doaj   +1 more source

Computational correction of copy-number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells

Nature Genetics, 2017
The CRISPR–Cas9 system has revolutionized gene editing both at single genes and in multiplexed loss-of-function screens, thus enabling precise genome-scale identification of genes essential for proliferation and survival of cancer cells.
R. Meyers, Jordan G. Bryan, James M. McFarland, B. Weir, Ann E. Sizemore, Han Xu, N. Dharia, Phillip G Montgomery, G. Cowley, S. Pantel, A. Goodale, Yenarae Lee, L. Ali, G. Jiang, Rakela Lubonja, W. Harrington, M. Strickland, Ting Wu, Derek Hawes, Victor A. Zhivich, M. Wyatt, Zohra Kalani, Jaime Chang, M. Okamoto, T. Golub, J. Boehm, F. Vazquez, D. Root, W. Hahn, Aviad Tsherniak   +29 more
semanticscholar   +1 more source

CRISPR/Cas9, a universal tool for genomic engineering

Вавиловский журнал генетики и селекции, 2016
The CRISPR/Cas9 system was initially described as an element of archeal and bacterial immunity, but gained much attention recently for its outstanding ability to be programmed to target any genomic loci through a short 20-nucleotide sgRNA region. Here we
A. V. Smirnov, A. M. Yunusova, V. A. Lukyanchikova, N. R. Battulin   +3 more
doaj   +1 more source