Vector shedding and immunogenicity sampling for retinal gene therapy

Abstract

There has been recent growth in the number and magnitude of clinical trials for various forms of retinal gene therapy. Because of regulatory requirements, and to better understand vector safety profiles, there is a need for standardised and effective methods to collect, process, and store biological samples taken from trial patients that can be used to assess the dissemination of the vector within bodily fluids and any systemic cellular and humoral immune responses.