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.

More information: Jiaming Wang et al, BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease, Science Translational ...

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

Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...

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 study by UMass Chan researchers showed that gene therapy correcting the mutation behind maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored coordinated gene ...

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, ...

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 ...