Hemophilia

Hemophilia A has been a prospective target for gene therapy since that field arose a quarter of a century ago. Recently, researchers at Yonsei University in South Korea have used the CRISPR-Cas9 system of gene editing to reverse a chromosome inversion of base pairs that cause this disease. The procedure applied to induced pluripotent stem cells in mice restored the normal production of clotting factor VIII. MORE

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