题目内容
Totreatthepatientswith Duchenne's muscular dystrophy,doctorsneedtocorrectthedefective________gene.
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Which of the following disease is the most commonly tested as an indication by gene therapy in patients?
About ____________ people have sickle cell disease in the US.
Worldwide, more than ____________ infants are born with sickle cell disease.
Spinal muscular atrophy (SMA) is a group of inherited disorders that cause progressive muscle degeneration and weakness, which is caused by gene mutations in the ________ and VAPB genes.
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