Objective
Inherited retinal degenerations are a primary clinical focus of adeno-associated virus (AAV) mediated gene therapy. These diseases mostly involve pathogenic mutations in the retinal pigment epithelium or photoreceptor transcripts leading to cell death and progressively lead to blindness. Current delivery methods are efficient for gene delivery to these cells as well as other therapeutically relevant cell types in the mouse retina. However the applicability of these vectors in human gene therapy depends on our ability to overcome the translational challenges between the mouse and human retinas. Naturally occuring AAVs efficient in transducing the mouse retina in most cases lead to insufficient expression in the primate retina due to interspecies differences in the patterns of transduction by AAV. To address the need for more performant AAVs for retinal gene delivery, we developed artificial AAV variants that have superior tranduction properties to the naturally occuring ones in the mouse retina. A resulting novel AAV variant mediated widespread gene delivery to the outer retina when injected into the vitreous in mice and in some areas of the primate retina. We would like to evaluate the therapeutic potential of this and other AAV variants based on this vector in the context of the primate retina. Our aim is to find suitable variants for application in three different gene therapy settings: gene replacement therapies aimed at the outer retina, trophic factor secretion by overexpression of neurotrophin genes and delivery of optogenetic tools to restrore light sensitivity to blind retinas after loss of vision.
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 microbiology virology
- medical and health sciences clinical medicine ophthalmology
- natural sciences biological sciences genetics mutation
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.
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-PEOPLE-2012-CIG
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.
MC-CIG - Support for training and career development of researcher (CIG)
Coordinator
75252 PARIS
France
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.