Treatment of Lysosomal Storage Diseases with Enzyme Replacement Therapy Involves

Multiple Choice

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