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:

Genomic Imprinting

A genetic phenomenon by which certain genes are expressed in a parent-of-origin-specific manner, meaning some genes are only active when inherited from either the mother or the father.

Placenta

An organ that develops in female mammals during pregnancy, providing oxygen and nutrients to the fetus while removing waste products.

Nutrients Diversion

The process where nutrients are redirected from one part of an organism to another, often to support growth, reproduction, or survival under stress.

Deleterious Effects

Harmful or damaging impacts, often used in the context of effects on health, environment, or genetic traits.