CAJ | 학술논문

视网膜疾病分子机制研究的不断深入,促进了视网膜疾病基因治疗的进展.腺相关病毒(AAV)的毒性和免疫原性均较低,外源基因表达稳定,可以转染多种分裂期和静止期细胞,因此成为治疗视网膜疾病的有效载体.在不同的动物模型和临床试验中,已有多项研究结果证实AAV作为载体治疗视网膜疾病的安全性和有效性.目前AAV在动物疾病模型中已经进行了介导抗血管生成蛋白、神经营养因子、抗凋亡因子的表达和基因替代治疗的研究,取得了令人满意的效果.但是,AAV也存在携带外源基因能力偏小的问题,需要进一步研究并解决之.
시망막질병분자궤제연구적불단심입,촉진료시망막질병기인치료적진전.선상관병독(AAV)적독성화면역원성균교저,외원기인표체은정,가이전염다충분렬기화정지기세포,인차성위치료시망막질병적유효재체.재불동적동물모형화림상시험중,이유다항연구결과증실AAV작위재체치료시망막질병적안전성화유효성.목전AAV재동물질병모형중이경진행료개도항혈관생성단백、신경영양인자、항조망인자적표체화기인체대치료적연구,취득료령인만의적효과.단시,AAV야존재휴대외원기인능력편소적문제,수요진일보연구병해결지.
Significant progress in understanding the molecular basis of retinal disorders has led to the development of gene therapies for treatment of these diseases.Adeno-associated virus (AAV) is a useful vector for the treatment of retinal diseases due to its low toxicity and dimmunogenicity,ability to transducer both dividing and non-dividing cells,and stable transgene expression.A variety of animal studies and clinical trials have proved the safety and effectivity of retinal AAV-mediated gene therapy.AAV-mediated gene therapy,such as anti-angiogenic proteins,neurotrophic factors,anti-apoptosis factors were studied in animal disease models,and the results were satisfactory.However,the main drawback of AAV vectors is its relatively small packaging capacity,which needs further improvement.