中国视网膜下基因治疗药物注射术操作规范推荐专家共识(2022)  

Expert consensus on surgical technique for subretinal injection of gene therapy in China(2022)

作　　者：中华医学会眼科学分会眼底病学组 中国医师协会眼科医师分会眼底病专业委员会 孙晓东[2] 许迅[2] 冯竞仰 吕林[3] 王雨生 魏文斌[5] 徐格致[6] 赵明威[7] Fundus Disease Group of Chinese Medical Association Ophthalmology Branch;Fundus Disease Committee of Chinese Medical Doctor Association Ophthalmology Branch;Sun Xiaodong;Xu Xun

机构地区：[1]不详 [2]上海交通大学附属第一人民医院,眼科国家眼部疾病临床医学研究中心 [3]中山大学中山眼科中心 [4]空军军医大学西京医院眼科,全军眼科研究所 [5]首都医科大学附属北京同仁医院眼科中心 [6]复旦大学附属眼耳鼻喉科医院 [7]北京大学人民医院眼科

出　　处：《中华实验眼科杂志》2022年第10期887-893,共7页Chinese Journal Of Experimental Ophthalmology

基　　金：上海市科学技术委员会项目(20Z11900400);上海申康医院发展中心临床科技创新项目(SHDC2020CR5014)。

摘　　要：随着2017年具有里程碑意义的Luxturna被美国食品药品监督管理局批准用于临床,基因治疗在眼科领域迅速发展。临床研究和实践表明,视网膜下注射术是遗传性视网膜疾病(IRDs)等眼底病变相关基因治疗药物有效的递送方法之一。视网膜下注射基因治疗是在实施玻璃体视网膜手术基础上叠加独特的给药操作,使基因治疗药物能够精准地聚积于目标区域并作用于靶细胞,而基因疗法的效果、病毒载体的分布、宿主组织的免疫反应程度均取决于药物能否被安全、高效地递送至视网膜下腔。我国IRDs等眼底病变患者众多,虽然基因治疗仍处于起步阶段,但已受到世界范围的高度关注,因此极有必要依据现有的国内外研究证据和临床实践,制定一个详细、完整的基因治疗药物视网膜下注射术操作流程专家推荐意见,以规范临床医生和研究者在相关疾病治疗过程中的应用,促进IRDs等眼底病变相关基因治疗临床研究的顺利开展和进步。未来随着基因治疗技术的发展,视网膜下注射术操作规范将不断完善更新。With the introduction of landmark genetic product,Luxturna,into clinical application in 2017,gene therapy is rapidly developed in ophthalmology.Clinical researches and practices have confirmed that subretinal injection technique is one of the important intraocular delivery approaches for gene therapy drugs related to inherited retinal diseases(IRDs)and other fundus diseases.The technique is characterized by the addition of unique drug delivery operation on the basis of vitreoretinal surgery,so that the therapeutic gene can precisely accumulate in the target area and act on the target cells.The efficacy of gene therapy,distribution of viral vector and degree of immune response mainly depend on the safe and efficient delivery of drugs into the subretinal space.There is a large number of patients with IRDs and other fundus diseases in China.Although gene therapy has just started,it has attracted great attention at home and overseas.Thus,it is necessary to develop a detailed and complete operative standard for subretinal injection of gene therapy according to the current research evidence and clinical practices worldwide.This expert consensus may standardize the surgical procedure of clinicians and facilitate the development of gene therapy related to IRDs and other fundus diseases.With the development of genetic technology,the relevant content will be constantly updated in future.

关 键 词：基因治疗/方法 人 眼内注射 视网膜疾病/遗传 视网膜疾病/治疗 光相干断层扫描 玻璃体切割术 操作规范 

分 类 号：R450[医药卫生—治疗学] R774[医药卫生—临床医学]