Treatment of lysosomal storage diseases with enzyme replacement therapy involves _______.
A)targeting functional copies of the missing enzyme to the precise cell sites where the deficiency is manifested
B)targeting mutant copies of the missing enzyme to the cell sites where the deficiency is manifested
C)targeting functional copies of another enzyme to the cell sites where the deficiency is manifested
D)administration of small molecular weight drugs to inhibit the synthesis of substances that accumulate in the disease
E)administration of large molecular weight drugs to inhibit the synthesis of substances that accumulate in the disease

Question

Quizplus · Accepted Answer

Enzyme replacement therapy involves administering functional copies of the missing enzyme to the precise cell sites where the deficiency is manifested. This allows for the enzyme to be taken up by the affected cells and improve their function by breaking down the accumulated substances that cause the lysosomal storage disease. Targeting mutant copies or unrelated enzymes would not be effective in treating the disease, and inhibiting synthesis of the accumulated substances would not address the underlying enzymatic deficiency.

Treatment of Lysosomal Storage Diseases with Enzyme Replacement Therapy Involves