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Virtual PhD defence: Andreas Braae Holmgaard

Fighting Retinal Diseases with RNA interference and CRISPR/Cas9 genome editing

2020.09.28 | Graduate School of Health

Date Fri 02 Oct
Time 14:00 16:00
Location Zoom

Friday 2 October at 14:00, Andreas Braae Holmgaard defends his PhD dissertation entitled “Fighting Retinal Diseases with RNA interference and CRISPR/Cas9 genome editing”.

The Vegfa growth factor is involved in the pathogenesis of age-related macular degeneration (AMD) and anti-VEGF therapy has been demonstrated to increase visual acuity in patients. As an alternative to repeated intraocular injections, we evaluate the potential of gene therapeutic targeting through Vegfa knockdown and knockout. We demonstrate that a multigenic expression cassette expressing PEDF and anti-VEGF shRNAs decrease VEGF expression in a CNV mice model. In order to expand the applicability of retinal genome editing, we evaluated whether insertions or deletions (idels) could be introduced into the Vegfa gene in RPE cells following delivery of SpCas9 by lentiviral vectors or lipoplexes. Following FACS isolation of eGFP-positive RPE cells, we demonstrate indel formation in the Vegfa gene using lentiviral vectors harboring three different sgRNAs. As well, we demonstrate that non-viral lipoplex-based retinal delivery of RNPs including chemically modified sgRNAs induce indels in the Vegfa gene, however, with lower efficacy. Consequently, we as the first demonstrate retinal indels using lentiviral delivery and using RNPs containing chemically modified sgRNAs.

The summary is written by the PhD student.  

The defence is public and will be held virtually via Zoom, https://aarhusuniversity.zoom.us/j/67702729132. Please read the attached press release for more information. 


PhD student Andreas Braae Holmgaard

Mail: anho@biomed.au.dk

Tel.: (+45) 28268737

Read full press release

PhD defense, Public/Media, Graduate School of Health, Graduate School of Health, PhD students, Department of Biomedicine, Academic staff, Health