Browsing FAS Scholarly Articles by Keyword "CRISPR/Cas9"

Now showing items 1-3 of 3

    • Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles 

      Wang, Ming; Zuris, John; Meng, Fantao; Rees, Holly Anne; Sun, Shuo; Deng, Pu; Han, Yong; Gao, Xue; Pouli, Dimitra; Wu, Qi; Georgakoudi, Irene; Liu, David Ruchien; Xu, Qiaobing (Proceedings of the National Academy of Sciences, 2016)
      A central challenge to the development of protein-based therapeutics is the inefficiency of delivery of protein cargo across the mammalian cell membrane, including escape from endosomes. Here we report that combining ...

    • 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 ...

    • Quality control, modeling, and visualization of CRISPR screens with MAGeCK-VISPR 

      Li, Wei; Köster, Johannes; Xu, Han; Chen, Chen-Hao; Xiao, Tengfei; Liu, Jun S.; Brown, Myles; Liu, X. Shirley (BioMed Central, 2015)
      High-throughput CRISPR screens have shown great promise in functional genomics. We present MAGeCK-VISPR, a comprehensive quality control (QC), analysis, and visualization workflow for CRISPR screens. MAGeCK-VISPR defines ...