Objective
Mutations in the Crumbs homologue 1 (CRB1) gene cause photoreceptor degeneration resulting in progressive retinitis pigmentosa (RP) or Leber congenital amaurosis (LCA), which both currently are untreatable blinding diseases. CRB1 is localized in Mueller glia cells at a subapical region (SAR) adjacent to adherens junctions between Mueller glia cells and photoreceptors. Loss of CRB1 function results in loss of adhesion between Mueller glia cells and photoreceptors. This ultimately results in the death of photoreceptors and other retinal neurons with loss of vision at birth (LCA) or before the age of 20 years (progressive RP). Using fruitfly and mouse genetics we will analyze the biochemical, cellular, developmental and physiological functions of CRB1, its family member CRB3, and CRB1-interacting-proteins PALS1, PATJ and MUPP1, in function of the SAR and its role in cell-cell interactions. We will analyze the effect of loss of interaction between Mueller glia cells and photoreceptors and subsequent retinal degeneration in retinas lacking both CRB1 and CRB3, or lacking the CRB1-interacting protein PALS1. To further understand the role of these proteins in neural-glial interactions in general, we will also assess what role these molecules play during development. To develop a cure restoring the impaired interaction between glial cells and photoreceptors, we will explore the efficacy of Mueller glia progenitor cell transplantation in mouse retinas. In collaboration with a small enterprise we will also deliver clinical grade adeno-associated viral (AAV) gene therapy vectors to transfer and express human CRB1 specifically in Mueller glia cells lacking CRB1 function. At the end of the project we will develop a demonstration project using these vectors in clinical phase I/II tests.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences biochemistry biomolecules proteins
- natural sciences biological sciences genetics mutation
- medical and health sciences clinical medicine transplantation
You need to log in or register to use this function
We are sorry... an unexpected error occurred during execution.
You need to be authenticated. Your session might have expired.
Thank you for your feedback. You will soon receive an email to confirm the submission. If you have selected to be notified about the reporting status, you will also be contacted when the reporting status will change.
Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-HEALTH-2007-A
See other projects for this call
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
1011 JV AMSTERDAM
Netherlands
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.