Myotonic Dystrophy Type 1 (MD1)

Myotonic Dystrophy Type 1 (MD1)

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...
Dilated Cardio Myopathy (DCM)

Dilated Cardio Myopathy (DCM)

Therapeutic area: Muscular Disorders, Cardiomyopathy Disease: Dilated Cardio Myopathy  Monogenic inroad: TITIN Cellular Model: Patient-derived cardiomyocytes (ventricular) Project Origin:   Technische Universität München, Klinikum rechts der Isar, Prof. Karl-Ludwig...
Schizophrenia

Schizophrenia

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...
Smarca2 in Cancer

Smarca2 in Cancer

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...
Parkinson’s Disease

Parkinson’s Disease

Therapeutic area: CNS, neurodegenerative disorders Disease: Parkinson’s Disease Monogenic inroad: Alpha Synuclein (SNCA) and LRRK2 (G2019S) Cellular Model: Patient-derived cell lines Project Origin: University of Luxembourg, Prof. Rejko Krüger Medical...