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