The Great Hope

The Great Hope

Doctors told him, that he would never be older than 19. Today, Ben Dupree is in his mid-twenties. But his body is continuously breaking down its own muscle tissue. In early stages it only affects the legs but it will inevitably reach his respiratory muscles and presumably his heart too. The reason is a single false letter within his genes – one letter out of billions in the human genome. As a result he suffers from a terminal illness known as Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy

Duchenne Muscular dystrophy is a common inherited muscular disorder – and it is always fatal. Patients lack the protein Dystrophin. As a result, muscle fiber degenerates over time. Even the respiratory muscles and usually also the heart muscles are affected. The reason: Duchenne-Patients suffer from a gene mutation, which prohibits the correct production of the protein Dystrophin. It leads to an incomplete and thus useless formation of the protein. The disease affects about one in 4000 boys. No cure has been found yet.

But now researchers have hope to put an end to the disease soon– with a new gene editing technology: CRISPR-Cas. Ben´s cells are the first cells whose genomes were repaired with the help of this new method - for the time being only in the laboratory. Ben hopes that their efforts will lead to a gene therapy, which could potentially prolong his live. However gene therapy is uncertain and risky.

Filmmaker: Dirk Gilson

Stand: 05.06.2017, 09:00