Funding decision
Applicant / Contact person	Malm, Tarja
Organisation	University of Eastern Finland
Project title	Multicellular organoids: modeling, mechanisms and therapy development for C9ORF72-associated neurodegeneration
Decision No.	334801
Decision date	16.12.2019
Funding period	01.01.2020 - 31.12.2022
Funding	150 917
Project description The delivery of therapeutic agents to the central nervous system remains a major challenge. This is especially true in neurodegenerative diseases. While significant discoveries have recently been made in the genetics of ALS/FTD, patients still have no real therapeutic treatments. Here we aim to boost the clinical translation of the preclinical research and assembled a multidisciplinary research team with complimentary expertise to (1) generate 3D in vitro multicellular organoid models from iPSCs derived from healthy and patients with ALS/FTD; (2) fully characterize the newly generated models using established assays in the consortium (e.g. molecular markers and electrophysiology); (3) explore mechanistic pathways in particular pro-inflammatory, anti-oxidant and DNA damage pathways and ultimately use the model for (4) drug and gene based therapeutic screening.