Objective
Skeletal dysplasias are a diverse and heterogeneous group of hereditary disorders characterized by malformations of bone and cartilage. These conditions affect development patterns in bones of growing children, and the clinical severity ranges from mild short stature to highly disfiguring, and even to lethal forms. Their treatment is very challenging, typically with no or limited success. This proposal is designed to explore the use of gene- and stem cell-based approaches to influence bone development in growth plate disorders characterized by aberrant constitutive cellular signaling in the growth plate chondrocytes. These biotherapeutic technologies offer the unique opportunity to manipulate and influence the cells in the developing growth plate, and if successfully applied, may functionally rescue bone growth and restore normal skeletal development. In this study, we will work to gain fundamental knowledge necessary for the rational design of a therapy for achondroplasia and Noonan syndrome. In attempting to address skeletal dysplasias, we have chosen to focus on these diseases because they are caused by gain-of-function point mutations affecting intracellular signaling in growth plate chondrocytes, and there exists transgenic mouse models containing mutations identical to that found in affected patients. The following specific aims will be addressed: Aim 1- To determine the biological effects of systemic gene delivery and overexpression of sFGFR3 in Fgfr3ach/+ mice. Aim 2- To determine if overexpression of sFGFR3 or inhibition of SHP2 expression can restore growth in bones from Ptpn11D61G/+ mice in an organ culture system. Aim 3- To determine the capacity of exogenous stem cells to influence the biology of diseased growth plate in vivo. The fellowship will contribute to the reintegration of Dr. Gouze, an expert in gene and stem cell therapies for musculoskeletal disorders, in the INSERM unit of Pr. Salles, an expert in pediatric endocrinology and growth disorders
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 cells technologies stem cells
- natural sciences biological sciences genetics mutation
- medical and health sciences clinical medicine endocrinology
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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-PEOPLE-IRG-2008
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
75654 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.