This is the first time human cells, engineered in this particular way, have been given back to a patient. The technology has got enormous potential to correct other conditions

2019-06-30: Germline Gene Therapy

means of correcting disease-causing nuclear and mitochondrial DNA mutations in gametes or preimplantation embryos have now been developed and are commonly referred to as germline gene therapy (GGT). We will discuss these novel strategies and provide a path forward for safe, high-efficiency GGT that may provide a promising new paradigm for preventing the passage of deleterious genes from parent to child.

2020-12-07: Cures

Both the Vertex/CRISPR and Bluebird techniques seem to work – and in fact, to work very well. There are now people walking around, many months after these treatments, who were severely ill but now appear to be cured. That’s not a word we get to use very often.

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