Correct Answer: A [A rare genetic disorder ]
Notes:
Scientists have developed a new gene therapy for Maple Syrup Urine Disease (MSUD), a rare
genetic disorder. MSUD is caused by a deficiency of the enzyme complex needed to break down
branched-chain amino acids (BCAAs) like leucine, isoleucine, and valine. It is inherited in
an autosomal recessive pattern, requiring both parents to pass on the gene mutation. The
disorder prevents proper amino acid metabolism, leading to neurological symptoms and
life-threatening brain damage.