Joint Transnational Call 2017 – Funded Projects

Joint Transnational Call (JTC2017)

  • CureDravet: Curing Dravet Syndrome by Gene Therapy
  • ERAAT: Enhancing Endoplasmic Reticulum Proteostasis to Rescue Alpha1 Antitrypsin Deficiency
  • Cure-AID: IL-18 and MRP neutralization for the treatment of anti-IL-1-refractory autoinflammatory diseases
  • SCA-CYP: Gene Therapy for Cerebellar Ataxias: restoring cholesterol metabolism by targeting brain cholesterol 24 hydroxylase (CYP46A1)
  • EDSCIDPROG: Gene edited lymphoid progenitors for adoptive transfer as a treatment of primary immunodeficiency
  • TREAT-HGPS: Exploring new therapeutic strategies in Hutchinson-Gilford progeria syndrome preclinical models
  • TreatOPON: Preclinical Development of Treatments for OPA1-linked Optic Neuropathies
  • TreatPolyQ: Allele-specific lowering of mutant polyQ proteins as treatment for Huntington disease, spinocerebellar ataxia type 3 and spinocerebellar ataxia type 7
  • MuTaEB : Mutation-targeted gene and pharmacological therapies for dystrophic and junctional Epidermolysis Bullosa
  • TREAT-MTMs: Novel therapies for neuromuscular diseases with altered phosphoinositide metabolism
  • CALSER: The effect of CDNF in ALS and ER stress