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 coordinated expression of the affected genes and stabilized biomarkers in a calf as well as in mice.
Study demonstrates successful gene therapy for maple syrup urine disease
- Post author:admin
- Post published:March 2, 2025
- Post category:uncategorized