Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

被引：54

作者：

Trapani, Ivana ^{[1
,2
]}

机构：

[1] Telethon Inst Genet & Med TIGEM, I-80078 Pozzuoli, Italy

[2] Univ Naples Federico II, Dept Translat Med, Med Genet, I-80131 Naples, Italy

来源：

GENES | 2019年 / 10卷 / 04期

关键词：

AAV; retina; gene therapy; dual AAV; PACKAGING CAPACITY; ANIMAL-MODELS; AAV VECTORS; MOUSE MODEL; VIRUS; EXPRESSION; THERAPY; TRANSDUCTION; SYSTEM; RECONSTRUCTION;

D O I：

10.3390/genes10040287

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Gene therapy using adeno-associated viral (AAV) vectors currently represents the most promising approach for the treatment of many inherited retinal diseases (IRDs), given AAV's ability to efficiently deliver therapeutic genes to both photoreceptors and retinal pigment epithelium, and their excellent safety and efficacy profiles in humans. However, one of the main obstacles to widespread AAV application is their limited packaging capacity, which precludes their use from the treatment of IRDs which are caused by mutations in genes whose coding sequence exceeds 5 kb. Therefore, in recent years, considerable effort has been made to identify strategies to increase the transfer capacity of AAV vectors. This review will discuss these new developed strategies, highlighting the advancements as well as the limitations that the field has still to overcome to finally expand the applicability of AAV vectors to IRDs due to mutations in large genes.

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页数：12

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