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Improving the efficiency of gene targeting in blood stem cells

As mentioned in the introductory post to this series on blood stem cell gene therapy, several significant issues existed with the first set of clinical trials in the gene therapy world. It became abundantly clear that simply delivering the gene target on its own would...

Obtaining more target cells for gene therapy

Blood stem cells are the fundamental component of regenerative medicine applications involving the blood and immune system. Despite significant efforts and investment, the research community has largely failed to maintain fully functional blood stem cells for...