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Content archived on 2024-05-29

Screening for peptides that revert mutant huntingtin induced abnormal phenotypes

Objective

The description of the initial molecular events in Huntington's disease is crucial to design therapeutic intervention for the cure of this devastating disorder. In this proposal, I plan to identify the early HD-trigger mechanism, aiming for the development of rational therapeutics. Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder due to an expanded, unstable CAG repeated sequence that elongates a segment of glutamine residues in the HD gene's protein product, huntingtin.

The disease is characterized by progressively worsening chorea, psychiatric impairment and cognitive decline. The most striking pathological manifestation of HD is a specific and gradual loss of neurons, predominantly in the caudate nucleus and putamen of the brain. I propose, via a combinatorial screening, to isolate small peptides that specifically bind mutant huntingtin and that are able to reverse in vivo pathogenic phenotypes by displacing crucial protein-protein interactions.

An aptamer with such characteristics will represent a very exciting lead to design small drugs for therapeutic intervention. I anticipate that this study will provide a better understanding of the first molecular events of the disease, providing suitable targets for therapeutic intervention.

Fields of science (EuroSciVoc)

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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.

Call for proposal

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FP6-2002-MOBILITY-12
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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.

IRG - Marie Curie actions-International re-integration grants

Coordinator

INTERNATIONAL SCHOOL FOR ADVANCED STUDIES
EU contribution
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Total cost

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