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Why has enzyme replacement therapy not been as effective for lysosomal storage d

ID: 162108 • Letter: W

Question

Why has enzyme replacement therapy not been as effective for lysosomal storage disorders that affect brain tissue?

The enzymes can't get across the blood brain barrier.

the enzymes usually break up in the acidic environment of the stomach

All of the above.

The enzymes are metabolized by the liver prior to them becoming effective

The enzymes a filter by the kidney too quickly to reach the brain

The enzymes can't get across the blood brain barrier.

the enzymes usually break up in the acidic environment of the stomach

All of the above.

The enzymes are metabolized by the liver prior to them becoming effective

The enzymes a filter by the kidney too quickly to reach the brain

Explanation / Answer

ans A

Lysosomal storage disorders are the group of disorders resulting from the deficiency of single acid hydrolase. Enzyme replacement therapy has not been successful to treat disorder affecting brain tissues as the enzymes cannot cross blood brain barrier.

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