Wednesday 6 August 2014

Gene Correction Without Mutating Off-Target Sites

Efficient integration of a corrected gene into a patient's genome without mutating off-target sites is a major hurdle in gene therapy. In a paper published today in emGenome Research/em, scientists have used CRISPR/Cas genome editing technology to seamlessly and efficiently correct disease-causing mutations in cells from patients with (and) #946;-thalassemia. (and) #946;-thalassemia results from inherited DNA mutations in the hemoglobin beta (HBB) gene, resulting in reduced ...

via Medindia Health News More READ

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