Selection of a novel AAV2/TNFAIP3 vector for local suppression of islet xenograft inflammation.

Nathan W ZammitKaren L SeebergerJad ZamerliStacey N WaltersLeszek LisowskiGregory S Korbutt Shane T Grey

Published in: Xenotransplantation (2020)

We report a new protocol that allows for high-efficiency genetic modification of NPIs, which can be utilized to introduce candidate genes without the need for germline engineering. This approach would be suitable for preclinical and clinical testing of beneficial molecules. We also report for the first time that A20 is cytoprotective for NPI, such that A20 gene therapy could aid the clinical development of NPIs for beta cell replacement.

Keyphrases

gene therapy
high efficiency
oxidative stress
randomized controlled trial
cell therapy
genome wide
stem cells
copy number
bone marrow