Sentences with phrase «for myotonic»

This is already underway for a compound whose corrective effect was observed in cells carrying a mutation responsible for myotonic dystrophy type I.

The proof of concept of such approach has been initiated in the team on a PGD - derived hES cells carrying the causative mutation for Myotonic Dystrophy type 1 (DM1).

Not exact matches

Validating this original concept, we previously demonstrated that PGD - derived hES cells and their derivatives, which express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), offer pertinent disease - cell models, applicable for a wide systemic mechanistic analysis ranging from functional studies at the cellular level to a large - scale drug screening.

For the first time, through the use of human embryonic stem cells (hES) sourced from pre-implantation diagnosis, researchers from Inserm's Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I - Stem) have successfully identified the previously unknown mechanisms involved in Steinert» disease, also known as type 1 myotonic dystropFor the first time, through the use of human embryonic stem cells (hES) sourced from pre-implantation diagnosis, researchers from Inserm's Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I - Stem) have successfully identified the previously unknown mechanisms involved in Steinert» disease, also known as type 1 myotonic dystropfor Stem Cell Therapy and Exploration of Monogenic Diseases (I - Stem) have successfully identified the previously unknown mechanisms involved in Steinert» disease, also known as type 1 myotonic dystrophy.

Validating this concept, we previously demonstrated that human pluripotent stem cells and derivatives which, express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), offer pertinent disease - cell models, applicable for a wide systemic analysis ranging from mechanistic studies to therapeutic screening.

CpG methylation, a parent - of - origin effect for maternal - biased transmission of congenital myotonic dystrophy.

Dr. Barbé: The molecular mechanisms responsible for the onset and severity of repeat diseases, such as myotonic dystrophy and Huntington's disease, are largely unknown.