Browsing FAS Scholarly Articles by Keyword "Cas9"
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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 ...