📋 Tender Overview
A Novel And Empowered Targeted Gene Addition Approach At A Relevant Microglia Locus For The Treatment Of Inherited Neurometabolic Diseases
Improved Hematopoietic Stem Cell Gene Therapy Technology To Treat Neurometabolic Disordersin The Last Decade, Gene Therapy, Including Hematopoietic Stem Cell (Hsc) Gene Therapy, Has Demonstrated Unprecedented Therapeutic Potential Thanks To Innovations In Vector Design, Stem Cell Manipulation, And Delivery. Recently There Has Been Strong Interest In The Development Of Hsc Gene Therapy Approaches For Neurometabolic Disorders (Nmd), Which Are Monogenetic Diseases Affecting The Lysosomal Or Peroxisomal Metabolic Activity. In These Disorders, Defects In The Nervous System Lead To Neuronal Death, Microglial Activation, Demyelination And Axonal Degeneration. The Eu-Funded Targetnmds Project Aims To Develop An Effective Treatment For Nmds Based On An Hsc Targeted Gene Addition Approach At A Newly Identified Microglia Locus. The Defect Correction At This Hsc Locus Could Generate A Microglia-Like Progeny With Unprecedented Therapeutic Potential.
Funded Under
European Research Council (Erc)
Total Cost
€ 2 495 250,00
Eu Contribution
€ 2 495 250,00
Coordinated By
Universita Degli Studi Di Padova
Italy
