CORDIS Project
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This project aims to develop a gene therapy for X-linked Retinitis pigmentosa, a severe eye disorder. The strategy involves using a viral vector to deliver DNA repair tools to promote the correction of genetic mutations in photoreceptors, potentially restoring vision in affected individuals.
In this project the applicant proposes to develop a treatment strategy for a devastating blinding disorder affecting photoreceptor function within the first decade of life, X-linked Retinitis pigmentosa (XLRP).
No treatment option exists to date.The proposed treatment strategy is based on the idea of inducing homology directed repair (HDR) of the mutation by promoting the exchange between the endogenous mutated chromosomal sequence and an exogenous repair DNA template at a double strand break (D…
JUSTUS-LIEBIG-UNIVERSITAET GIESSEN
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