Browsing Harvard Medical School by Keyword "Cas9"
Now showing items 1-5 of 5
-
Characterization and Optimization of the CRISPR/Cas System for Applications in Genome Engineering
(2014-07-07)The ability to precisely manipulate the genome in a targeted manner is fundamental to driving both basic science research and development of medical therapeutics. Until recently, this has been primarily achieved through ... -
The Clustered, Regularly Interspaced, Short Palindromic Repeats-associated Endonuclease 9 (CRISPR/Cas9)-created MDM2T309G Mutation Enhances Vitreous-induced Expression of MDM2 and Proliferation and Survival of Cells
(American Society for Biochemistry & Molecular Biology (ASBMB), 2016)The 309G allele of single nucleotide polymorphisms (SNPs) in the mouse double minute (MDM2) promoter locus is associated with a higher risk of cancer and proliferative vitreoretinopathy (PVR), but as to whether this SNP ... -
Engineering microdeletions and microduplications by targeting segmental duplications with CRISPR
(2016)Recurrent, reciprocal genomic disorders resulting from non-allelic homologous recombination (NAHR) between near-identical segmental duplications (SDs) are a major cause of human disease, often producing phenotypically ... -
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 ... -
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
(2016)Current genome-editing technologies introduce double-stranded (ds) DNA breaks at a target locus as the first step to gene correction.1,2 Although most genetic diseases arise from point mutations, current approaches to point ...