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 MSN14d
Gene Therapy for Maple Syrup Urine Disease: A Promising New ApproachMSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to break down certain parts of proteins. This ...
New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.
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