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Logical control of CRISPR gene editing system
Oxford University
Gene therapy
Genetic disorders
Target Identification or Validation
CRISPR is described as “the technology of the century” and brings the hope of treatments for currently incurable diseases, by excising or silencing genes that drive tumor proliferation, repair of faulty genes, or by killing diseased cells. However, unwanted DNA cuts, resulting from nonspecific delivery to off-target cells, remain a serious problem in the successful clinical translation of CRISPR based therapeutics. Exact spatio-temporal control of CRISPR/Cas9 activity would lead to the development of new therapies and new tools for biotechnology with great translational potential.