Publication in the journal Molecular Therapy by the team of Jocelyn Laporte at IGBMC
Further work from Jocelyn Laporte’s laboratory confirming that ‘turning down’ DNM2 (one of the genes that causes CNM) is an effective therapeutic approach to myotubular myopathy. Their work so far has also confirmed that down-regulating DNM2 is curative for the myopathy caused by the genes BIN1, DNM2 and MTM1.
In this new study, published in the journal Molecular Therapy, Drs Cowling and Tasfaout tested another approach, using a viral vector (AAV) to deliver the same result. This further confirms the DNM2 down-regulation route to a treatment for different forms of myotubular and centronuclear myopathy. Myotubular Trust thanks our many supporters who make it possible for us to be able to help fund this excellent research.