摘要
遗传性耳聋是常见的致残性疾病之一,临床尚无有效治疗药物.近年,基因治疗在临床前遗传性耳聋动物模型中获得显著疗效,推动了多项耳聋基因治疗临床试验的启动和开展.然而,目前尚无针对耳聋基因治疗的相关规范或共识.复旦大学附属眼耳鼻喉科医院作为国际首个成功开展遗传性耳聋基因治疗临床试验的单位,积累了一定经验.基于此,并参考其他疾病基因治疗共识,联合上海市多家单位的耳科学、听力学、遗传学、基因治疗及康复学等专家,在适用人群选择、围手术期准备、基因治疗药物注射及其安全性和有效性评估、治疗后康复等方面制订本共识,为相关从业人员提供参考和依据,以规范遗传性耳聋基因治疗的临床开展.
Abstract
Hereditary hearing loss is one of the common disabling diseases.Up to now,there are no available drugs for clinical therapy.Recently,gene therapy has achieved an obvious therapeutic effect in animal models with hereditary hearing loss,promoting the initiation and development of several clinical trials of gene therapy for hearing loss.However,there is currently no relevant standard or consensus on gene therapy for hearing loss.Eye&ENT Hospital of Fudan University has first successfully carried out clinical trials of gene therapy for hereditary hearing loss in the world,accumulating a certain amount of experience.On this basis,with reference to the consensus on gene therapy for other diseases,we cooperated with experts in otology,audiology,genetics,gene therapy,and rehabilitation,etc.,in Shanghai,and conducted this consensus on the applicable population,perioperative preparation,drug delivery of gene therapy,safety and effectiveness evaluation,and post-treatment rehabilitation,etc,providing reference and basis for relevant practitioners,standardizing the clinical treatment of gene therapy for hereditary hearing loss.
基金项目
国家杰出青年科学基金(82225014)
国家重点研发计划合成生物学重点专项(2020YFA0908201)
国家自然科学基金面上项目(82171148)
上海市"科技创新行动计划"生物医药科技支撑专项(2021)(21S11905100)
NETL
NSTL
维普
万方数据