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find Keyword "Delivery route" 1 results
  • Research progress of effect of different delivery routes of adeno-associated virus on retinal gene therapy

    At present, 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. There are currently three ways to deliver AAV vector-mediated target genes to the retina. 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. Moreover, studies on X-linked retinoschisis have been undergoing clinical trials, but intravitreal injection has poor transduction in retinal pigment epithelium (RPE) cell layer and photoreceptor cell (PR) layer. Subretinal injection of AAV vector can deliver the target gene to the local retina, resulting in stronger efficiency of transfection and gene expression in the RPE and PR layers, which has been undergoing in animal and clinical trials, 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, which is less invasive than intravitreal and subretinal injection, Suprachorioidal injection is an important delivery route for future exploration of AAV vector mediated gene therapy for retinal diseases.

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