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Question

Which enzyme was targeted during the first clinical gene therapy given in 1990 to a four year- old girl?

A
MAO
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B
ADA
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C
Tyrosine oxidase
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D
Pyruvate dehydrogenase
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Solution

The correct option is B ADA
The first clinical gene therapy was given in 1990 to a four- year- old girl with adenosine deaminase (ADA deficiency). This enzyme is crucial for the immune system to function because in its absence lymphocytes proliferation is inhibited.

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