The first trial to edit genes in patients with Danon Disease

Rare genetic disorder caused by mutations in LAMB2 gene will receive the first gene therapy attempt. The trial started recruitment and is available at ClinicalTrials.gov.

Danon Disease manifests itself by weakening of muscles (including heart) and intellectual disability. Male carriers survive 19 years on average.

Genetic correction offers obvious improvement of life or even possible cure to the disorder. Scientists from Rocket Pharmaceuticals will use virus particles containing healthy LAMB2 gene, with a goal of restoring its function in the body.

The trial will consist of just single injection – probably because of unclear consequences. We can expect, that successful correction of essential tissues (especially: heart muscle) will need more or even different methods of administration.