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Maple Syrup Urine Disease
March 7 , 2025
29 days
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- The Scientists have created a new gene therapy for a debilitating genetic disorder called maple syrup urine disease (MSUD).
- The treatment can prevent recurrence of deadly symptoms in a cow calf born with the disease.
- Classic MSUD arises from mutations in the three genes that encode the protein subunits of branched chain alpha-keto acid dehydrogenase complex (BCKDH).
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