Currently, the only cure for alpha thalassemia is a bone marrow transplant, but the success of gene therapies for related conditions points to a potential new cure.
On a quest to bring new a new hemophilia gene therapy to the clinic, one research team leapt over hurdle after hurdle to produce enough viral therapeutics to treat a large number of patients.
Recent advancements in mitochondrial genome editing technologies take scientists one step closer to developing viable treatments for mitochondrial diseases, which affect 1 in 4300 adults.
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