MSUD 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 ...

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.

New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.