Name of Fellow

    RUI JORGE GONÇALVES PEREIRA NOBRE

    Institution

    Funder

    FCT

    Contact information of fellow

    Country

    Portugal

    Title of project/programme

    SILENCING MACHADO-JOSEPH DISEASE THROUGH THE SYSTEMIC ROUTE

    Source of funding information

    FCT

    Total sum awarded (Euro)

    € 116,640

    Start date of award

    01/01/10

    Total duration of award in years

    6.0

    The project/programme is most relevant to:

    Spinocerebellar ataxia (SCA)

    Keywords

    Research Abstract

    Our group has recently showed that allele-specific silencing of ataxin-3 significantly decreased the severity of the neuropathological abnormalities in a rat model of Machado-Joseph disease (MJD) upon intracranial injection of lentiviral vectors (Alves et al., 2008a). Although intracranial injection is a safe procedure there is a need for a less invasive system. The aim of this project is to develop a system that will allow delivery of siRNA-based treatments to the brain by a vascular route. AAV9 vectors will be engineered to transduce neurons of the adult mouse. Silencing sequences, previously shown to efficiently abrogate MJD, will be cloned into AAV9 and its efficacy evaluated in animal models (Alves et al., 2008b). It is expected that this project will enable the development of a new gene delivery system for the treatment of MJD and constitute a proof of principle for systemic gene therapy of a wide range of neurodegenerative diseases.

Types: Fellowships
Member States: Portugal
Diseases: Spinocerebellar ataxia (SCA)
Years: 2016
Database Categories: N/A
Database Tags: N/A

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