Sentences with phrase «dystrophy type»
The phrase "dystrophy type" refers to a specific category or group of muscle disorders that cause weakness and deterioration in the body's muscles. Full definition
This is already underway for a compound whose corrective effect was observed in cells carrying a mutation responsible for myotonic dystrophy type I.
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ORDO is used for «naming» diseases e.g. «autosomal recessive limb girdle muscular dystrophy type 1», while HPO is used for describing the clinical phenotype observed in a patient e.g. «muscle weakness».
However, due to a work accident in 2008, her handler developed a neurological condition known as Reflex Sympathetic Dystrophy type II with Dystonia.
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.
Graduate students, from left, Long Luu and Lien Nguyen, with University of Illinois chemistry professor Steven Zimmerman, developed drug compounds that target three pathways associated with myotonic dystrophy type 1.
«Drugs with multiple targets show promise against myotonic dystrophy type 1.»
Efforts to treat myotonic dystrophy type 1, the most common form of muscular dystrophy, are in their infancy.
These repeats, called CTG expansions in myotonic dystrophy type 1, become «toxic» when transcribed from DNA.
A group of researchers has shown for the first time in cells and in a mouse model that a drug used to treat cancer can neutralize the toxic RNA that causes the prolonged muscle contractions and other symptoms of myotonic dystrophy type 1, the most common form of adult - onset muscular dystrophy.
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.
As a proof of principle, we demonstrated that PGD - derived hES cells and derivatives which, express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), may mimic molecular defects associated to the pathology.
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).
Four research teams of I - Stem have joined forces in a collaborative project that has just achieved a first pilot therapy - oriented screen of compounds and RNA interference aiming at reversing the altered phenotypes observed in human embryonic stem cells carrying the mutant gene for myotonic dystrophy type 1.
A defective Krab - domain zinc - finger transcription factor contributes to altered myogenesis in myotonic dystrophy type 1.
mTOR - dependent proliferation defect in human ES - derived neural stem cells affected by myotonic dystrophy type 1.
We demonstrated the proof of this concept by identifying new genes participating to the Myotonic Dystrophy type 1 (DM1) pathogenesis.
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.