"Current animal experiments show that healthy genes can be successfully introduced to replace defective genes. The problem is that after outside genes are introduced, they are attacked by the patient's own immune cells, so that their therapeutic effect is maintained for only around six months before weakening and then disappearing," Chen explains, admitting that there is still a long way to go before gene therapy for Pompe disease is available.
「目前動物實驗已經可以成功地置入健康基因以取代缺損的基因，問題是外來基因置入後會遭到患者自體免疫細胞的攻擊，大約維持 6 個月左右就弱化而消失不見了，」陳垣崇坦承，龐貝氏症的基因治療還有一段路要走。