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Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Anesthesia, Children's Hospital Boston, Boston, MA, USA;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,;
Department of Pediatrics,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Powell Gene Therapy Center,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,|Powell Gene Therapy Center,;
Department of Pediatrics,|Department of Surgery, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA and;
Department of Pediatrics,;
Department of Physical Therapy and;
Department of Physical Therapy and;
Department of Physical Therapy and;
Department of Pediatrics,|Powell Gene Therapy Center,;
机译:生成蜂窝模型研究基因治疗术治疗POPPE病
机译:36个月后随访评估在后期发病Pompe疾病中停止和恢复酶替代疗法:来自瑞士Pompe登记处的数据
机译:AAV介导的POPPE疾病基因治疗的进步
机译:Gaa的下一代测序的微流体PCR扩增以检测引起群众疾病的突变
机译:皮博迪发育运动量表2在接受酶替代疗法的庞贝病婴儿和儿童中的并发有效性和反应性
机译:庞培疾病遗传分析的两种方法:基于一般人群的基因组数据分析的患者文献综述
机译:酶替代疗法在成人中停止酶促毒性:评估欧洲POMPE联盟停止标准