题目内容

Totreatthepatientswith Stargardt disease,doctorsneedtocorrectthedefective________gene.

ABCA4
B. ABCB4
C. ABCC4
D. ABCD4

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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

Worldwide, more than ____________ infants are born with sickle cell disease.

A. 250,000
B. 260,000
C. 275,000
D. 300,000