Gene editing and its application for hematological diseases.

  title={Gene editing and its application for hematological diseases.},
  author={Mark J. Osborn and Joseph J. Belanto and Jakub Tolar and Daniel F. Voytas},
  journal={International journal of hematology},
  volume={104 1},
The use of precise, rationally designed gene-editing nucleases allows for targeted genome and transcriptome modification, and at present, four major classes of nucleases are being employed: zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), meganucleases (MNs), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9. Each reagent shares the ability to recognize and bind a target sequence of DNA. Depending on the properties of the reagent… CONTINUE READING
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High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide offtarget effects

  • BP Kleinstiver, V Pattanayak, MS Prew
  • Nature
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