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Most single-gene diseases, such as hemophilia, are caused by different mutations scattered in a specific gene rather than a single predominant mutation, so the team needed to develop a vector that would be applicable for patients with any mutations. The study is a preclinical proof of concept using a universal CRISPR/Cas9 gene targeting approach that could be applied to majority of the patients with a specific disease, in this case hemophilia B. According to the Centers for Disease Control and Prevention, hemophilia in general occurs in approximately 1 in 5,000 live births and there are about 20,000 people with hemophilia in the United States.
good news if it works out
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