Rescue Of Misfolded Proteins In Rare Diseases By Using Known Molecules

Universities and Institutes of Italy


September 27, 2021


  • Organisation/Company: Università degli Studi di Padova
  • Research Field: Biological sciences
  • Researcher Profile: First Stage Researcher (R1) Recognised Researcher (R2) Established Researcher (R3) Leading Researcher (R4)
  • Application Deadline: 27/09/2021 23:59 - Europe/Brussels
  • Location: Italy
  • Type Of Contract: To be defined
  • In sarcoglycanopathies, a structural defect (misfolding), caused by a point mutation, results in the elimination of a protein (sarcoglycan) with the consequent loss of function. To avoid the elimination of the mutants, we propose the second use of small molecules, developed for cystic fibrosis and known as CFTR correctors. With this project we intend to: finalize the in vivo (on sarcoglycanopathy mouse model) validation of the efficacy of the most promising compound; set the best drug regimen; test other correctors (especially those already in clinic for cystic fibrosis); evaluate the effect of co-administration, since in vitro data indicate a synergistic action of some correctors Data del bando 05/08/2021

    Eligibility criteria

    Eligible destination country/ies for fellows:

  • Italy
  • Eligibility of fellows: country/ies of residence:

  • Eligibility of fellows: nationality/ies:

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    Contact Information
  • Organisation/Company: Università degli Studi di Padova
  • Organisation Type: Public Research Institution
  • Website: https:// www.
  • Country: Italy
  • City: Padova