Snyder Richard O, Flotte Terence R
Powell Gene Therapy Center, University of Florida Genetics Institute, Department of Pediatrics, 1600 SW Archer Road, Gainesville, FL 32610-0266, USA.
Curr Opin Biotechnol. 2002 Oct;13(5):418-23. doi: 10.1016/s0958-1669(02)00369-5.
Recombinant adeno-associated viral (rAAV) vectors are being evaluated in animal models and humans. Pre-clinical data demonstrating vector safety, efficiency and efficacy have been used to initiate human clinical trials. The clinical manufacture of rAAV vectors has supported phase I and phase II trials, showing that adeno-associated virus serotype 2 vectors are safe when administered to humans.
重组腺相关病毒(rAAV)载体正在动物模型和人类中进行评估。证明载体安全性、效率和有效性的临床前数据已被用于启动人类临床试验。rAAV载体的临床生产支持了I期和II期试验,表明腺相关病毒2型载体在给人类使用时是安全的。