Beam Therapeutics launched BEAM-304, a new program using base editing technology to treat phenylketonuria (PKU), aiming to correct mutations in the PAH gene for patients with this rare metabolic disorder. This development is significant as it could reduce toxic phenylalanine levels and potentially eliminate lifelong dietary restrictions for PKU sufferers, highlighting the potential of genetic medicine to address unmet medical needs.
Read the full article at Genetic Engineering News
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