Deep molecular phenotyping of myotonic dystrophy (DM1) hiPSC-cardiomyocytes to facilitate risk stratification and drug evaluation
Professor Chris Denning and colleagues at the University of Nottingham will use stem cells to investigate how to treat a genetic condition that often leads to death from abnormal heart rhythms, known as myotonic dystrophy. They will take skin cells from patients with myotonic dystrophy and turn them into stem cells in the laboratory. They will then direct the stem cells to develop into heart cells, which can be closely studied and used to test treatments including gene and drug therapies. Myotonic dystrophy type 1 (DM1) is an incurable condition that often leads to an early death from abnormal heart rhythms. The genetic cause is known: it is associated with extra genetic material in a gene called DMPK. Studies in the lab using cells or animals have helped understand DM1. However, no-one has investigated the condition in functioning human heart cells and little is known as to why the heart is badly affected. The researchers have considerable experience in making stem cells from patients with genetic conditions and converting these stem cells into heart cells. Their research will help develop treatments for people with myotonic dystrophy.
Project details
| Grant amount | £323,626 |
|---|---|
| Grant type | Project Grants |
| Application type | Project Grant |
| Start Date | 01 May 2015 |
| Duration | 3 years |
| Reference | PG/14/59/31000 |
| Status | Complete |
