NIH Symposium Explores Promise of Stem Cell TherapiesMonday, Jul 14, 2008
Stem cells have been hailed as a toolkit to treat a host of diseases, but at an NIH symposium on May 6, researchers said they
are still deciphering the toolkit’s instruction manual.
New Gene Therapy Vector May Lead to Treatment for Muscular DystrophyMonday, Apr 18, 2005
One of the biggest challenges in developing useful gene therapy is finding a way to get the beneficial gene into enough cells
of the body to effectively treat the disease. Now, researchers have shown in rodents that a virus called adeno-associated
virus 8 (AAV8) can effectively deliver a gene to all the skeletal muscles of the body. If it works the same way in humans,
this virus-based approach may allow the first effective gene therapy for muscular dystrophy (MD) and similar diseases.
New Gene Therapy Vector May Lead to Treatment for Muscular DystrophyMonday, Apr 18, 2005
One of the biggest challenges in developing useful gene therapy is finding a way to get the beneficial gene into enough cells
of the body to effectively treat the disease. Now, researchers have shown in rodents that a virus called adeno-associated
virus 8 (AAV8) can effectively deliver a gene to all the skeletal muscles of the body. If it works the same way in humans,
this virus-based approach may allow the first effective gene therapy for muscular dystrophy (MD) and similar diseases.
Scientists Identify a New Kind of Genetic Problem in Muscular DystrophyThursday, Aug 8, 2002
A newly identified genetic problem underlies a common neuromuscular disorder called facioscapulohumeral muscular dystrophy
(FSHD), scientists say. In a new study, they show that deletion of repetitive DNA sequences in people with this disorder
allows nearby genes to go into overdrive. The finding solves a decade-old riddle about the cause of this disorder and may
ultimately lead to the first effective treatments.
Fact Sheet Faulty Muscle Repair Implicated in Muscular DystrophiesWednesday, May 21, 2003
Researchers have revealed what may be a totally new cause for muscular dystrophy (MD). A recent study shows that a protein
defective in two types of late-onset MD plays a critical role in the normal repair of muscles.
Fact Sheet A New Test for Myotonic Dystrophy: Exposing an Enemy That’s Too Big to SeeWednesday, Mar 26, 2003
Researchers have developed a genetic test that detects a common form of muscular dystrophy with 99 percent accuracy. The
accurate diagnosis of myotonic muscular dystrophy type 2 (DM2) allows researchers to fully describe its clinical features
for the first time.
Fact Sheet Accurate and Affordable Diagnosis of Duchenne Muscular DystrophyFriday, Apr 18, 2003
Researchers have developed a simple and affordable blood test that detects the most common form of muscular dystrophy (MD)
in more than 95 percent of cases.
Fact Sheet Gene "Knockouts" Reveal Critical Links in Synapse FormationThursday, May 16, 1996
New studies reveal exciting clues to the mystery of how synapses form between nerve and muscle cells. The findings shed new
light on human development and may help reveal how molecular interactions are altered in muscular dystrophy.
Faulty Muscle Repair Implicated in Muscular DystrophiesWednesday, May 21, 2003
Researchers have revealed what may be a totally new cause for muscular dystrophy (MD). A recent study shows that a protein
defective in two types of late-onset MD plays a critical role in the normal repair of muscles.
Fact Sheet Accurate and Affordable Diagnosis of Duchenne Muscular DystrophyFriday, Apr 18, 2003
Researchers have developed a simple and affordable blood test that detects the most common form of muscular dystrophy (MD)
in more than 95 percent of cases.
Fact Sheet A New Test for Myotonic Dystrophy: Exposing an Enemy That’s Too Big to SeeWednesday, Mar 26, 2003
Researchers have developed a genetic test that detects a common form of muscular dystrophy with 99 percent accuracy. The
accurate diagnosis of myotonic muscular dystrophy type 2 (DM2) allows researchers to fully describe its clinical features
for the first time.
Fact Sheet Scientists Identify a New Kind of Genetic Problem in Muscular DystrophyThursday, Aug 8, 2002
A newly identified genetic problem underlies a common neuromuscular disorder called facioscapulohumeral muscular dystrophy
(FSHD), scientists say. In a new study, they show that deletion of repetitive DNA sequences in people with this disorder
allows nearby genes to go into overdrive. The finding solves a decade-old riddle about the cause of this disorder and may
ultimately lead to the first effective treatments.
Fact Sheet Scientists Identify Gene for Spinocerebellar Ataxia 2Thursday, Oct 31, 1996
Scientists have identified the gene altered in one of the most common hereditary ataxias, spinocerebellar ataxia 2 (SCA2).
The discovery allows improved genetic testing and provides new clues about how genetic mutations cause several neurological
disorders, including Huntington's disease. The findings are reported by three different groups in the November issue of Nature
Genetics.
Gene "Knockouts" Reveal Critical Links in Synapse FormationThursday, May 16, 1996
New studies reveal exciting clues to the mystery of how synapses form between nerve and muscle cells. The findings shed new
light on human development and may help reveal how molecular interactions are altered in muscular dystrophy.
New Type of Trinucleotide Mutation Found in Friedreich's AtaxiaThursday, Mar 7, 1996
Scientists have identified a new type of trinucleotide repeat mutation that leads to Friedreich's ataxia (FA), a rare childhood
neurodegenerative disease. The discovery allows accurate screening for carriers of the disease and may lead to the first effective
treatments.
NINDS Scientists Develop Strategy To Speed Gene and Brain ResearchThursday, Jun 20, 1991
Using a novel strategy, scientists at the National Institute of Neurological Disorders and Stroke (NINDS) have isolated key
identifying regions of more than 400 genes that work inside the human brain. The scientists say their work should help identify
genetic defects that cause brain disease and speed progress of genetics research.