New advances in gene therapy with adenoviral vectors for inherited retinal diseases

Q4 Medicine

中华眼底病杂志 Pub Date : 2020-03-25 DOI:10.3760/CMA.J.CN511434-20190705-00214

Kejiong Shen

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引用次数: 0

Abstract

Adeno-associated viral vector (AAV) is the most important viral tool and has been widely used in gene therapy. Because of its small size, non-enveloped, non-pathogenic and other characteristics, so it is one of the main means to treat hereditary retinal diseases. Aiming at MERTK for retinitis pigmentosa, ND4 for Leber hereditary optic neuropathy or RPE1 for choroideremia, AAV gene therapy improved half patients' visual acuity in clinic tests. Besides, there are some clinic tests in progress for Leber’s congenital amaurosis, X-linked retinoschisis, Achromatopsia, age-related macular degeneration. But more researches need to be found before clinic test for Stargardt disease, Usher syndrome and nanophthalmos. At present, AAV gene therapy is mainly used for recessive hereditary retinal diseases, and technology is needed to intervene for dominant retinal diseases. For the treatment of hereditary retinal diseases, this will be an important and complex systematic project, which requires more human and material resources to participate in and study together, and we expect to have a great breakthrough in the near future. Key words: Retinal diseases/genetics; Gene therapy; Review; Adeno-associated virus

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腺病毒载体基因治疗遗传性视网膜疾病的新进展

腺相关病毒载体（AAV）是最重要的病毒载体，已广泛应用于基因治疗。由于其体积小、无包膜、无致病性等特点，是治疗遗传性视网膜疾病的主要手段之一。针对MERTK治疗视网膜色素变性、ND4治疗Leber遗传性视神经病变或RPE1治疗脉络膜炎，AAV基因治疗在临床测试中提高了一半患者的视力。此外，Leber先天性黑蒙、X连锁视网膜分裂、色盲、年龄相关性黄斑变性的一些临床测试正在进行中。但在对Stargardt病、Usher综合征和纳米邻苯二甲酸进行临床测试之前，还需要进行更多的研究。目前，AAV基因治疗主要用于隐性遗传性视网膜疾病，显性视网膜疾病需要技术干预。对于遗传性视网膜疾病的治疗来说，这将是一项重要而复杂的系统工程，需要更多的人力物力参与和共同研究，我们期待在不久的将来取得重大突破。关键词：视网膜疾病/遗传学；基因治疗；评审；腺相关病毒

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来源期刊

中华眼底病杂志 Medicine-Ophthalmology

CiteScore

0.40

自引率

0.00%

发文量

5383

期刊介绍： Chinese Journal of Ocular Fundus Diseases is the only scientific journal in my country that focuses on reporting fundus diseases. Its purpose is to combine clinical and basic research, and to give equal importance to improvement and popularization. It comprehensively reflects the leading clinical and basic research results of fundus disease disciplines in my country; cultivates professional talents in fundus disease, promotes the development of fundus disease disciplines in my country; and promotes academic exchanges on fundus disease at home and abroad. The coverage includes clinical and basic research results of posterior segment diseases such as retina, uveal tract, vitreous body, visual pathway, and internal eye diseases related to systemic diseases. The readers are medical workers and researchers related to clinical and basic research of fundus diseases. According to the journal retrieval report of the Chinese Institute of Scientific and Technological Information, the comprehensive ranking impact factor and total citation frequency of the Chinese Journal of Ocular Fundus Diseases have been among the best in the disciplines of ophthalmology, otolaryngology, and ophthalmology in my country for many years. The papers published have been included in many important databases at home and abroad, such as Scopus, Peking University Core, and China Science Citation Database (CSCD).