Research progress of effect of different delivery routes of adeno-associated virus on retinal gene therapy
10.3760/cma.j.cn511434-20230918-00391
- VernacularTitle:腺相关病毒不同递送途径对视网膜基因治疗影响的研究进展
- Author:
Bo WAN
1
;
Zibing JIN
Author Information
1. 首都医科大学附属北京潞河医院眼科，北京 101199
- Keywords:
Adeno-associated virus;
Delivery route;
Retinal gene therapy;
Review
- From:
Chinese Journal of Ocular Fundus Diseases
2024;40(5):409-414
- CountryChina
- Language:Chinese
-
Abstract:
The treatment of hereditary retinopathy depends on gene replacement or editing therapy, and adeno-associated virus (AAV) vector is one of the most widely used gene transfer vectors. The delivery methods of AAV vector-mediated target genes to the retina inlucde intravitreal injection, subretinal injection, and suprachorioidal injection. Intravitreal injection of AAV vector is currently the most commonly used delivery route, which can effectively improve the functions of retina disorders such as blinding retinal dystrophy in mice. Subretinal injection of AAV vector can deliver the target gene to the local retina, resulting in stronger efficiency of transfection and gene expressio, however, the high technical operations are required. In recent years, as a new high-profile delivery route suprachorioidal injection of AAV vector can achieve more extensive transfection of target genes in the retina of rabbits and rats. At present, the efficiency of AAV vector transduction in the retina is affected by the delivery mode. In the future, it is necessary to further explore the effect of AAV vector delivery mode on the transduction efficiency in order to find an important delivery route for mediating gene therapy for retinal diseases.
