Treatment of Lysosomal Storage Diseases with Enzyme Replacement Therapy Involves

question 117

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

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Definitions:

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