Objective
The ability to target gene expression with nucleic acid-based therapeutics including small interfering RNAs (siRNAs) has opened up novel treatment opportunities for genetic skin disorders. Although siRNAs have exquisite selectivity and potency, translation to the clinic has been hampered due to the lack of efficient delivery systems that allow penetration through the outer barrier of the skin, the stratum corneum, and uptake by the live cells of the dermis and epidermis. A number of physical and chemical methods have been used to deliver siRNA across the stratum corneum into the epidermis. Once in the tissue, the target cells must internalize the siRNA. “Self-delivery” siRNAs have been identified that obviate the need for transfection reagents and are widely used in vitro. However, cellular uptake of the most effective self-delivery siRNAs is still inefficient compared to siRNA complexed with transfection reagents. Other functionalities may enhance delivery, but screening large numbers of modifications for self-delivery properties is hindered by cumbersome RNA synthesis methods. The long-term goal of this project is to develop novel siRNA conjugates that will facilitate optimal delivery to the affected tissues and cells for treatment of genetic disorders, particularly those of the skin. Here, it is proposed to develop an efficient method for conjugating siRNAs to a library of small molecules. The proposed strategy takes advantage of standard thiol and amine chemistries, producing a stable, covalent crosslink between the siRNA and any molecule containing a primary amine. These conjugates will initially be screened using a keratinocyte cell line expressing a reporter gene. In preparation for translation to the clinic, lead inhibitors will be fully characterized and tested for their ability to specifically inhibit reporter and endogenous gene expression in vitro and in vivo.
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: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- natural sciences biological sciences genetics RNA
- natural sciences chemical sciences organic chemistry amines
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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-2013-IIF
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
DD1 4HN Dundee
United Kingdom
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.