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Contenuto archiviato il 2024-05-14

Cloning of the friedreich ataxia gene and characterization of the encoded protein

Obiettivo



Friedreich ataxia (FRDA) is a severe neurodegenerative autosomal recessive disorder which affects the central and peripheral nervous system. It has an incidence of about 1 in 50000 in European populations.
As the primary biochemical defect is not known, positional cloning represents the best option for the identification of the defective gene. The combination of rare recombination(s) analysis and of gene exclusion by absence of causal mutation allowed us to narrow down the FRDA gene to a 300 kb interval. The objectives of the project are:
1. Search for the FRDA gene. 41 potential exons were isolated from a contig of overlapping cosmids by three different approaches, exon trapping prediction from random sequencing and cDNA selection. These potential exons will save to identify genes in the critical region by RACE PCR, exon connection and cDNA library screening, and will allow to search for mutations in patients in order to identify the Friedreich ataxia gene. 2. Unravelling the FRDA protein function. I will produce monoclonal antibodies against the FRDA protein, which will be used for the cellular and subcellular localisation of the FRDA protein. I will also initiate the construction of a mouse animal model for the disease, as this model will serve to understand the physiopathology of the disease and to test various therapeutic approaches.

Campo scientifico (EuroSciVoc)

Programma(i)

Argomento(i)

Invito a presentare proposte

Dati non disponibili

Meccanismo di finanziamento

RGI - Research grants (individual fellowships)

Coordinatore

Centre National de la Recherche Scientifique
Contributo UE
Nessun dato
Indirizzo
1,Rue Laurent Fries 1
67404 Illkirch
Francia
Costo totale
Nessun dato

Partecipanti (1)