Dystrophies in Biological News

Sticky protein helps reinforce fragile muscle membranes

... "The findings may also have clinical implications for muscular dystrophies that are caused by abnormal dystroglycan." These congenital muscular dystrophies include Fukuyama Congenital Muscular Dystrophy, Walker-Warburg Syndrome ...

March of Dimes awards $250,000 prize to scientists unraveling the causes of muscular dystrophy

... as well as childhood conditions such as Duchenne and Becker muscular dystrophies and congenital muscular dystrophy. The March of Dimes Prize is a ... Campbell and Dr. Kunkel have provided crucial insights into how muscular dystrophies develop the critical step needed to develop diagnostic tools and ...

Massive microRNA scan uncovers leads to treating muscle degeneration

... Institute, was senior investigator. The disorders include the muscular dystrophies (Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle ... the discoveries into treatments. For instance, most muscular dystrophies begin with a known mutation in a master gene, leading to damaged or absent ...

Stabilizing force for good communication between neurons and muscle cells found

... receptor apparently interact, then hsp90β comes along to help stabilize the relationship. Rapsyn mutations have been implicated in muscular dystrophies including congenital myasthenia gravis. MCG researchers are looking now to see if a mutated rapsyn still interacts with hsp90β. They used a ...

New clinical trial results show how personalized medicine will alter treatment of genetic disorders

... patients with Duchenne muscular dystrophy," writes Dr. Hoffman. "But in order to realize the promise of personalized molecular medicine in muscular dystrophies and, ultimately, other disorders, it will be important to re-evaluate current measures of toxicity, efficacy, and marketing that ensure both safety ...

Scientists characterize proteome of human cornea

... fungal, and viral infections (keratitis) and allergies to various dystrophies including keratoconus. "Corneal damage and disorders account for ... of inherited corneal disorders including granular and lattice corneal dystrophies are characterized by deposition of insoluble and opaque macromolecules in ...

Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

... of Molecular Therapy (now available online). "Gene therapy in muscular dystrophies presents a unique challenge, because replacement of deficient, therapeutic ... therefore apply to other forms of muscular dystrophy, they added. Muscular dystrophies include many genetic diseases, all of which are characterized by ...

Gene therapy reverses genetic mutation responsible for heart failure in muscular dystrophy

... into muscle cells in a fairly wide area around the injection site. However, for gene therapy treatments to be successful, particularly for muscular dystrophies where many organs and tissues are affected throughout the body, intramuscular injection is not practical for delivering a corrective gene to the ...

No small feat: First ever gene therapy success for muscular dystrophy achieved

... treatment can have expansive reach to muscles throughout the body and significantly increase survival. CMD is a group of some 20 inherited muscular dystrophies characterized by progressive and severe muscle wasting and weakness first noticed soon after birth. No effective treatments exist and children ...

Chemical 'band-aid' prevents heart failure in mice with muscular dystrophy

... corrective effect in the mdx mice, which really surprised us." In future research, U-M scientists want to test P188 on mice with other types of dystrophies and see if it works as well when given as injections, rather than infusions. Metzger emphasizes that it is uncertain whether P188 will have any ...