Focus on CRISPR tools and therapies
CRISPR-based genome editing has become a mainstay of experimental biology and is now maturing into a tool for therapeutic applications. Constant progress is being made in designing ever more sophisticated protein machines to catalyze desired changes with high efficiency and specificity, and in the methods to deliver the macromolecules involved to the tissues and cells. Many of the more recent advances have not entered clinical development yet, but will ultimately help therapeutic genome editing fulfill its full potential. Illustration of the structure of the CRISPR–Cas9 gene-editing complex, showing the guide RNA template (red), the Cas9 protein (blue) and the double strand of DNA (gray).
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