HSØ iPS-ALS: Bridging from iPS cells to clinical trials for Norwegian ALS patients in an international setting
The main aim of this proposal is to exploit the unique opportunity of iPS cell technology to derive patient-specific MNs and astrocytes carrying ALS-causing mutations to address key questions about autonomous and non-cell autonomous mechanisms of MN degeneration. The proposal will involve the generation of iPS cells from genetically characterized Norwegian ALS patients, which will then be differentiated into astrocytes and MNs in collaboration with top international experts. The ALS patient-derived MNs and astrocytes will be interrogated for molecular and physiological properties relevant to disease progression (morphogenesis, viability, inter- and intracellular signaling). In addition, the project will exploit recent advances in gene editing using the CRISPR/Cas9 platform to attempt to correct mutations in ALS patient-derived iPS cells, thus generating non-diseased, patient-specific autologous MNs and astrocytes that can be used both as controls in experimental mechanistic studies and potentially as cell treatment options. Lastly, the project will promote the development in Norway of neurosurgical and other clinical expertise necessary to establish satellite clinical trials in Norway linked to larger clinical trials in other countries. Thus, the project will for the first time bring Norwegian ALS patients into the sphere of preclinical and clinical studies aimed at developing life-saving treatments.
- Lagret: Room 2268
- Biobanknummer: 511200-007
- Ansvarlig: Joel Glover
REK - Ja 1 fil
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