Browsing FAS Scholarly Articles by Keyword "Cas9"

Now showing items 1-3 of 3

    • Engineering microdeletions and microduplications by targeting segmental duplications with CRISPR 

      Tai, Derek J. C.; Ragavendran, Ashok; Manavalan, Poornima; Stortchevoi, Alexei; Seabra, Catarina M.; Erdin, Serkan; Collins, Ryan L.; Blumenthal, Ian; Chen, Xiaoli; Shen, Yiping; Sahin, Mustafa; Zhang, Chengsheng; Lee, Charles; Gusella, James F.; Talkowski, Michael E. (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 

      Ott de Bruin, Lisa M.; Volpi, Stefano; Musunuru, Kiran (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 

      Komor, Alexis C.; Kim, Yongjoo B.; Packer, Michael S.; Zuris, John A.; Liu, David R. (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 ...