Monday, 22 February 2016

» Parent Project Italy Conference – Research Updates Action Duchenne

» Parent Project Italy Conference – Research Updates Action Duchenne: "Last weekend the Action Duchenne team attended the Italian Parent Project conference in Rome. The first session concentrated on pre-clinical updates, of which an interesting session was on CRISPR genome editing and DMD. The technique is called CRISPR and is a “cut and sew gene” that allows you to edit and correct the DNA with a potential, precision and versatility. The mechanism of this “molecular scissors”is based on the combination of few elements: the Cas9 protein, which is an enzyme capable of cutting the DNA, and a guide RNA, molecule designed ad hoc in the laboratory, which directs the cut of Cas9 in a very specific point, for example at the level of a mutation. This approach has seen many recent advances and could potentially tackle duplications and large deletions in the dystrophin gene, but, much more needs to be understood, for example off-target effects of making changes directly at the DNA level."

'via Blog this'

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