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Knut Stieger, DVM, Ph.D. - "Insights into in vivo genome editing in the retina - Focus on DNA repair mechanisms"

Department of Ophthalmology, Faculty of Medicine, Justus-Liebig-University Giessen, Germany
When Nov 13, 2018
from 10:00 AM to 11:30 AM
Where Tigem, Vesuvius Auditorium
Contact Name
Contact Phone 081-19230659
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Abstract
Inherited retinal dystrophies are a group of disorders which lead to visual impairment and, most severely, to blindness. The advent of genomic editing technologies such as CRISPR-Cas9 has opened new possibilities to target genes at endogenous genomic loci. However, gene editing requires maximum precision of DNA repair to avoid de-novo mutations at the target site.
It turned out that there is insufficient knowledge of the DNA repair machinery in postmitotic neurons, specifically in rod and cone photoreceptors. Recent studies even suggest a DNA repair deficiency being present in murine rod photoreceptors. Consequently, efficient therapeutic DNA repair has so far not been achieved, and it remains to be seen, whether this will ever become reality.
In order to shed further light on the status of DNA repair in photoreceptors, we studied the gene expression profile of DNA repair proteins in vivo in several different model systems. Furthermore, we have developed a number of different reporter gene systems and mouse models to develop therapeutic applications and to improve DNA repair efficacy.
In this seminar I will summarize our current data on in vivo DNA repair mechanisms and discuss future options on how to realize efficient therapeutic applications.

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