Navigation Links
Researchers discover target that could ease spinal muscular atrophy symptoms
Date:1/7/2009

COLUMBIA, Mo. There is no cure for spinal muscular atrophy (SMA), a genetic disorder that causes the weakening of muscles and is the leading genetic cause of infant death, but University of Missouri researchers have discovered a new therapeutic target that improves deteriorating skeletal muscle tissue caused by SMA. The new therapy enhanced muscle strength, improved gross motor skills and increased the lifespan in a SMA model.

"This therapy does not directly target the disease-causing gene; instead it targets the pathways that affect muscle maintenance and growth," said Chris Lorson, investigator in the Christopher S. Bond Life Sciences Center and associate professor of veterinary pathobiology in the MU College of Veterinary Medicine. "We administered a particular protein, follistatin, to SMA mouse models to determine if enhanced muscle mass impacts the symptoms of SMA. After treatment, the mice had increased muscle mass, gross motor function improvement and an increase in average life span of 30 percent."

With the therapy, MU researchers inhibited myostatin, a protein that limits muscle tissue growth. Myostatin activity can be reduced significantly by enabling several proteins that bind to myostatin, including follistatin. When myostatin is inhibited, muscle mass and strength increase.

SMA is caused by the loss of survival motor neuron-1(SMN1). Humans have a nearly identical copy gene called SMN2. Because of a single molecular difference, SMN2 alone cannot compensate for the loss of SMN1.

"While most work in the SMA field has logically focused on targeting the SMN2 gene, the results of this study suggest that skeletal muscle is a viable therapeutic target that may reduce the severity of some SMA symptoms," said Lorson, who also is the scientific director for FightSMA, a private spinal muscular atrophy research foundation in Richmond, Va. "Because follistatin does not alter the expression level of SMN protein, the most effective treatment would combine strategies that directly address the genetic defect in SMA as well as SMN-independent strategies that enhance skeletal muscle."


'/>"/>

Contact: Kelsey Jackson
JacksonKN@missouri.edu
573-882-8353
University of Missouri-Columbia
Source:Eurekalert

Related medicine news :

1. Health-monitoring technology helps seniors live at home longer, MU researchers find
2. Old gastrointestinal drug slows aging, McGill researchers say
3. NC State researchers find new bartonella species that infects humans
4. Researchers Report Progress in Fight Against Fat
5. Mayo researchers offer new insight into effectiveness of procedure to stop heavy menstrual bleeding
6. Burnham researchers illuminate mechanisms that regulate DNA damage control and replication
7. Relocation plan of metastatic cancer cells uncovered by Stanford researchers
8. Researchers at Columbia University Medical Center link blood sugar to normal cognitive aging
9. Researchers create smaller, brighter probe tailored for molecular imaging and tumor targeting
10. Nutritious fast-food kids meals are scarce, researchers find
11. UIC researchers hunting drugs for devastating parasitic disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/26/2016)... Lake Orion, Clarkston, Michigan (PRWEB) , ... June ... ... direction with respect to fertility once they have been diagnosed with endometriosis. These ... tolerable intercourse but they also require a comprehensive approach that can help for ...
(Date:6/25/2016)... TX (PRWEB) , ... June 25, 2016 , ... ... the United States, named Dr. Sesan Ogunleye, as the Medical Director of its new ... the facility Medical Director of our new Mesquite location,” said Dr. James M. Muzzarelli, ...
(Date:6/24/2016)... ... , ... A recent article published June 14 on E Online ... on to state that individuals are now more comfortable seeking to undergo not only ... calf and cheek reduction. The Los Angeles area medical group, Beverly Hills Physicians (BHP) ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... Global law ... magazine’s 2016 Legal Elite. The attorneys chosen by their peers for this recognition are ... , Seven Greenberg Traurig Shareholders received special honors as members of this year’s Legal ...
(Date:6/24/2016)... ... June 24, 2016 , ... ... Clinical Decision Making in Emergency Medicine conference in Ponte Vedra Beach, FL. The ... published in Emergency Medicine Practice and Pediatric Emergency Medicine Practice. , ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... , June 23, 2016 Research ... MEMS Devices Medical Market Analysis 2016 - Forecast to 2022" ... The report contains up to date financial data ... analysis. Assessment of major trends with potential impact on the ... analysis of market segmentation which comprises of sub markets, regional ...
(Date:6/23/2016)... Bracket , a leading clinical trial technology ... outcomes platform, Bracket eCOA (SM) 6.0, at the 52 ... 30, 2016 in Philadelphia , Pennsylvania.  A ... product of its kind to fully integrate with RTSM, will ... 6.0 is a flexible platform for electronic clinical outcomes assessments ...
(Date:6/23/2016)... -- Astellas today announced the establishment of Astellas Farma Colombia (AFC), a new affiliate with operations headquartered in ... . ... ... ... Astellas is ...
Breaking Medicine Technology: