Like research on gene therapies for other diseases, research for such a treatment for Pompe disease faces the twin dilemmas of short persistence times and poor therapeutic effectiveness after genes are introduced into the patient's body.
和其他疾病的基因治療研究相同，龐貝氏症也是卡在基因殖入患者體內後「存活不久」和「表現不佳」的兩大難題上。