Authors
Creative Biogene
Summary
Adeno-associated virus (AAV) is one of the most active vectors for gene therapy. The trials of gene therapy mediated by AAV vectors have been performed for the treatment of multiple diseases, including cancer and monogenic, cardiovascular, neurological, ocular, and infectious diseases. The milestone of AAV-mediated clinical studies is the development of inherited retinal diseases and anerythrochloropsia gene therapy. AAV-mediated PRE65 gene expression efficiently recovered visual function in patients with Leber's congenital amaurosis with controlled safety and efficacy of gene transfer. These encouraging results broaden the clinical applications of AAV vectors, including gene therapy of cancer.
Introduction
In cancer treatment, ideal gene transfer as well as expression should be restricted to the cell-type of interest such as the malignant cell or distinct cells of the tumor microenvironment like dendritic cells (DC), macrophages, endothelial cells or fibroblasts. Moreover, it would be advantageous to optimize efficacy to minimize the vector dose that needs to be applied and to be equipped for a possible re-application scenario. To achieve these goals, both the capsid and the genome of AAV vectors have become targets for engineering. In particular, transcriptional and post-transcriptional targeting, as well as transductional targeting strategies, have been developed.
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