Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
复制标题
DOI：
10.1089/humc.2014.2514
复制
发表时间：
2014-09-01
影响因子：
--
通讯作者：
Collins, Shelley W.
中科院分区：
文献类型：
--
作者：
Byrne, Barry J.;Smith, Barbara;Collins, Shelley W.
研究目的：评估膈肌施用重组腺相关病毒基因载体rAAV 1-CMV-GAA（标记为rAAV 1-p43. 2-hGAA），在庞贝氏症和呼吸机依赖的儿童。将研究两个剂量水平（1.0× 10 12和5.0× 10 12 vg）。
Study Objectives: Assessment of the safety of diaphragm administration of a recombinant adeno-associated virus gene vector, rAAV1-CMV-GAA (labeled as rAAV1-p43. 2-hGAA), in children with Pompe disease and ventilator dependence. Two dose levels (1.0× 10 12 and 5.0× 10 12 vg) will be studied.