Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.
腺相关病毒介导的α-葡萄糖苷酶基因疗法治疗庞贝氏症慢性呼吸衰竭的I/II期试验：初步安全性和通气结果
期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2012.250
Smith Barbara K, Collins Shelley W, Conlon Thomas J, Mah Cathryn S, Lawson Lee Ann, Martin Anatole D, Fuller David D, Cleaver Brian D, Clément Nathalie, Phillips Dawn, Islam Saleem, Dobjia Nicole, Byrne Barry J
