EX VIVO GENE THERAPY FOR FAMILIAL HYPERCHOLESTEROLEMIA

自1997年7月以来，11名儿童研究参与者在CHOP接受了重新评估。 未入组新的儿童受试者。 研究人员预计在明年招募一名新的儿童受试者。一名儿童研究参与者在常规评估期间发生了不良事件。 1998年8月12日，一名11岁的女性研究参与者在CHOP接受了心导管插入术。 她的左乳内动脉在手术过程中夹层，随后她有一个冠状动脉旁路移植术进行了当晚。 她恢复得很好。 该参与者不是接受体外基因治疗的五名参与者之一。研究团队已在HUP启动了成人受试者的脂蛋白动力学研究。 参与本研究是可选的，与年度常规评价同时进行。 几名成年人参与了研究，研究人员目前正在评估初步数据。

Since July of 1997, 11 child study participants have been re-evaluated at CHOP. No new child subjects have been enrolled. The investigators expect to enroll one new child subject during the coming year. One child study participant experienced an untoward event during her routine evaluation. On August 12, 1998 an 11 year old female study participant underwent a cardiac catheterization at CHOP. Her left internal mammary artery dissected during the procedure and subsequently she had a coronary artery bypass graft perfomed that evening. She has recovered well. This participant is not one of the five participants who received ex vivo gene therapy. The study team has initiated a lipoprotein kinetic study for the adult subjects at HUP. Participation in this study is optional and is conducted concurrently with the annual routine evaluation. Several adults have participated and the preliminary data are currently being evaluated by the investigators.