Seamless gene correction of beta-thalassemia mutations in patient-specific cells

5 agosto 2014

A major hurdle in gene therapy is the efficient integration of a corrected gene into a patient’s genome without mutating off-target sites. In a new paper, scientists explain having used CRISPR/Cas genome editing technology to seamlessly and efficiently correct disease-causing mutations in cells from patients with beta-thalassemia.
http://feeds.sciencedaily.com/~r/sciencedaily/~3/PgrZDPbs0zk/140805131957.htm

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