Journal article
AAV capsid bioengineering in primary human retina models
A Westhaus, SS Eamegdool, M Fernando, P Fuller-Carter, AA Brunet, AL Miller, R Rashwan, M Knight, M Daniszewski, GE Lidgerwood, A Pébay, A Hewitt, G Santilli, AJ Thrasher, LS Carvalho, A Gonzalez-Cordero, RV Jamieson, L Lisowski
Scientific Reports | Published : 2023
Abstract
Adeno-associated viral (AAV) vector-mediated retinal gene therapy is an active field of both pre-clinical as well as clinical research. As with other gene therapy clinical targets, novel bioengineered AAV variants developed by directed evolution or rational design to possess unique desirable properties, are entering retinal gene therapy translational programs. However, it is becoming increasingly evident that predictive preclinical models are required to develop and functionally validate these novel AAVs prior to clinical studies. To investigate if, and to what extent, primary retinal explant culture could be used for AAV capsid development, this study performed a large high-throughput scree..
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Awarded by Ophthalmic Research Institute of Australia
Funding Acknowledgements
This work was supported by project grants from the Australian National Health and Medical Research Council (NHMRC) to L.L. (APP1108311, APP1156431 and APP1161583) and Paediatrio Paediatric Precision Medicine Program to L.L. and A.G.-C. (PPM1 K5116/RD274). L.L. was also supported by research grants from the National Science Centre, Republic of Poland (OPUS 13) (UMO-2017/25/B/NZ1/02790). A.G.-C. was also supported by the MRFF Stem Cell Therapies Mission (2021/MRF2008912). A.J.T. was supported by funding from The Wellcome Trust (grant no. 217112/Z/19/Z AJT). This work was supported by research grants from the Ophthalmic Research Institute of Australia, the Sydney Research Excellence 2020 Initiative and Cure Blindness Australia to R.V.J. This work was supported by a Lions Eye Institute Strategic grant to L.S.C. A.P. was supported by a NHMRC Senior Research Fellowship (APP1154389).