Therapeutic area: Neuromuscular Disorders Disease: Myotonic Dystrophy Type 1 (MD-1) Monogenic inroad: DMPK Cellular Model: Patient derived skeletal muscle cells Project Origin: I-Stem, Cécile Martinat and Prof Marc Peschanski Medical need: MD1 is a type of muscular...
Therapeutic area: CNS, Psychiatric Disorders Disease: Schizophrenia Monogenic inroad: DISC1 Cellular Model: Patient derived glutamatergic neurons Project Origin: ZI Mannheim, Prof. Andreas Meyer-Lindenberg Medical need: Schizophrenia is a chronic and severe mental...
Therapeutic area: Oncology Disease: Non Small Cell Lung Cancers (NSCLC) / other cancers Monogenic inroad: Smarca 2 Cellular Model: Patient-derived cell line Project Origin: DKFZ, Prof. Nicolas Gunkel Medical need: SMARCA4 mutations have been identified in a high...
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