Results 31 to 40 of about 194,133 (208)
Knockdown of human AMPK using the CRISPR-Cas9 genome-editing system [PDF]
Methods Mol Biol, 1732, pp.171-194, 2018, 2019 AMP activated protein kinase (AMPK) is a critical energy sensor, regulating signaling networks involved in pathology including metabolic diseases and cancer. This increasingly recognized role of AMPK has prompted tremendous research efforts to develop new pharmacological AMPK activators.
arxiv +1 more source
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 +3 more
semanticscholar +1 more source
Science Advances, 2022 CRISPR-Cas9 gene editing has emerged as a powerful therapeutic technology, but the lack of safe and efficient in vivo delivery systems, especially for tissue-specific vectors, limits its broad clinical applications.
Tao Wan +5 more
semanticscholar +1 more source
Delivering CRISPR: a review of the challenges and approaches
Drug Delivery, 2018 Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR ...
Christopher A. Lino +3 more
doaj +1 more source
A small PAM optimises target recognition in the CRISPR-Cas immune system [PDF]
arXiv, 2021 CRISPR-Cas is an adaptive immune mechanism that has been harnessed for a variety of genetic engineering applications: the Cas9 protein recognises a 2-5nt DNA motif, known as the PAM, and a programmable crRNA binds a target DNA sequence that is then cleaved.
arxiv
Off- and on-target effects of genome editing in mouse embryos
The Journal of Reproduction and Development, 2018 Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas-based genome editing technology has enabled manipulation of the embryonic genome.
Shinya AYABE +2 more
doaj +1 more source
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 +25 more
semanticscholar +1 more source
Concerning RNA-guided gene drives for the alteration of wild populations
eLife, 2014 Gene drives may be capable of addressing ecological problems by altering entire populations of wild organisms, but their use has remained largely theoretical due to technical constraints. Here we consider the potential for RNA-guided gene drives based on
Kevin M Esvelt +3 more
doaj +1 more source
Rationally engineered Cas9 nucleases with improved specificity
Science, 2015 Making the correct cut The CRISPR/Cas system is a prokaryotic immune system that targets and cuts out foreign DNA in bacteria. It has been adopted for gene editing because it can be designed to recognize and cut specific locations in the genome.
I. Slaymaker +5 more
semanticscholar +1 more source
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing
Nature Genetics, 2020 Genome editing has therapeutic potential for treating genetic diseases and cancer. However, the currently most practicable approaches rely on the generation of DNA double-strand breaks (DSBs), which can give rise to a poorly characterized spectrum of ...
Mitchell L. Leibowitz +8 more
semanticscholar +1 more source