Treatment of Lysosomal Storage Disorders with Substrate Reduction Therapy Involves

question 32

Multiple Choice

Treatment of lysosomal storage disorders with substrate reduction therapy involves _______.

• A)targeting functional copies of the missing enzyme to the 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 the substances that accumulate in the disease
• E)administration of large molecular weight drugs to inhibit the synthesis of the substances that accumulate in the disease

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Definitions:

Heart Disease

A broad term that encompasses various conditions affecting the heart's structure and function, leading to significant health challenges and mortality risks.

Diabetes

A group of diseases that result in too much sugar in the blood (high blood glucose), the most common being type 1 and type 2 diabetes.

Polymorphisms

The occurrence of two or more clearly different forms, or alleles, within the population of a species.

Polytypic

Describing the expression of genetic variants in different frequencies in different populations of a species.