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