Sentences with phrase «myotonic dystrophy type»

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

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.

We demonstrated the proof of this concept by identifying new genes participating to the Myotonic Dystrophy type 1 (DM1) pathogenesis.

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).

mTOR - dependent proliferation defect in human ES - derived neural stem cells affected by myotonic dystrophy type 1.

Current development - stage programs include antisense drugs to treat SMA, ISIS - SMNRx, and myotonic dystrophy type 1, ISIS - DMPKRx.

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.

Because the anticancer compound works by binding to troublesome expansions in DNA, the researchers decided to test whether it would work in cells and a mouse model of myotonic dystrophy type 1.

Efforts to treat myotonic dystrophy type 1, the most common form of muscular dystrophy, are in their infancy.

«Drugs with multiple targets show promise against myotonic dystrophy type 1.»

Not exact matches

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 dystrophy.

Muscular dystrophies are an inherited group of disorders, and include Duchenne and Becker, Emery Driefuss, limb - girdle, myotonic, facioscapulohumeral, and distal types.

7000 compounds were tested on human mesenchymal stem cells carrying Type 1 myotonic dystrophy.