Treatment of lysosomal storage diseases with enzyme replacement therapy involves _______.
A)sending 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 sending functional copies of the missing enzyme to the precise cell sites where the deficiency is manifested in order to alleviate the symptoms and slow the progression of the disease. This allows the deficient cells to carry out their normal functions and reduce the accumulation of substrates. Targeting mutant copies of the missing enzyme or administering drugs to inhibit the synthesis of substances that accumulate in the disease are not part of enzyme replacement therapy.

Treatment of Lysosomal Storage Diseases with Enzyme Replacement Therapy Involves