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Projects. Life and Earth Sciences

Systemic analysis of the gene regulatory networks involved in the specification and maintenance of the retinal pigment epithelium: towards new therapies for retinal degenerative diseases

Lead Researcher: Paola Bovolenta Nicolao
Research Centre: Centro de Biología Molecular Severo Ochoa. CSIC-UAM. Madrid.

Abstract: 

Paola Bovolenta NicolaoThe human retina has a very limited regenerative potential and therefore cell therapy strategies are currently seen as a promising avenue for an efficient treatment of the most common retinal degenerative diseases, including Retinitis Pigmentosa. These diseases are characterized by a primary loss of photoreceptors and retinal-pigmented epithelium (RPE), a fundamental tissue for photoreceptors physiology. Therefore, cell therapies for vision recovery require considering the survival of both photoreceptors and RPE. A recurring obstacle to obtaining pigmented precursors from different stem cells sources derives from the limited knowledge we still have on the genetic networks that confer RPE identity and that maintain photoreceptor homeostasis and visual pigments recycling. This project aims at overcoming these obstacles by identifying key genes in RPE specification and maintenance. We will combine the zebrafish versatile genetic toolkit with the TRAP methodology (Translating Ribosome Affinity Purification), which allows in vivo transcriptomic analysis of targeted cell types by using tissue-specific transgenic lines. Using TRAP, we intend to characterize the RPE transcriptome during its specification and in response to photoreceptors damage. The most promising genes identified in transcriptomic studies will be further investigated by generating mutant zebrafish lines using the CRISPr/Cas9 technology. These mutants will constitute an important resource as may represent new animal models for retinitis pigmentosa that could be used in large-scale drug screenings, given the experimental advantages of zebrafish for systematic drug trials. In summary, our RPE view on retinal degeneration should open new conceptual spaces for gene and cell therapies in regenerative medicine.


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