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Gene Therapy for Cerebellar Ataxias: restoring cholesterol metabolism by targeting brain cholesterol 24 hydroxylase (CYP46A1) – SCA-CYP
Mutation-targeted gene and pharmacological therapies for dystrophic and junctional Epidermolysis Bullosa – MuTaEB
Enhancing Endoplasmic Reticulum Proteostasis to Rescue Alpha1 Antitrypsin Deficiency – ERAAT
Gene edited lymphoid progenitors for adoptive transfer as a treatment of primary Immunodeficiency – EDSCIDPROG
Curing Dravet Syndrome by Gene Therapy – CureDravet
Allele-specific lowering of mutant polyQ proteins as treatment for Huntington disease, spinocerebellar ataxia type 3 and spinocerebellar ataxia type 7 – TreatPolyQ
Preclinical Development of Treatments for OPA1-linked Optic Neuropathies – TreatOPON
Novel therapies for neuromuscular diseases with altered phosphoinositide metabolism – TREAT-MTMs