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.
Researchers are using CRISPR gene editing to restore function of the mutant protein that causes Duchenne muscular dystrophy, a genetic disease that causes muscle degeneration.
A native Hawaiian, anthropologist, and genome scientist, Keolu Fox investigates how mutations found in indigenous populations contribute to human health and emphasizes the importance of considering who benefits from research involving indigenous genomes.
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