A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
Medical Xpress on MSN19d
Study demonstrates successful gene therapy for maple syrup urine diseaseA 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 ...
Researchers in the current study designed a dual-function recombinant adeno-associated virus serotype 9 vector to deliver a gene replacement to the liver ... in two mouse models and a calf ...
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