More information: Jiaming Wang et al, BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

Gene therapy targeting maple syrup urine disease (MSUD) has shown potential in preventing newborn death, normalizing growth, and stabilizing biomarkers in animal models, including calves and mice.

“BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.” “Simply put, we believe the gene therapy ...

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 ...

A study led by UMass Chan Medical School researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, ...