题目内容

Totreatthepatientswith X-linked adrenoleukodystrophy (ALD),doctorsneedtocorrectthedefective________gene.

ABCA1
B. ABCB1
C. ABCC1
D. ABCD1

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Totreatthepatientswith Stargardt disease,doctorsneedtocorrectthedefective________gene.

ABCA4
B. ABCB4
C. ABCC4
D. ABCD4

Totreatthepatientswith Duchenne's muscular dystrophy,doctorsneedtocorrectthedefective________gene.

ABCA4
B. ABCD1
C. dystrophin
D. β-globin

Which of the following disease is the most commonly tested as an indication by gene therapy in patients?

A. infectious diseases
B. cancers
C. monogenic diseases
D. cardiovascular diseases

About ____________ people have sickle cell disease in the US.

A. 70,000
B. 80,000
C. 90,000
D. 100,000

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