December 18, 2017
Researchers on Tail of Treatment for Form of Muscular Dystrophy
Timothy Birdnow
Some good news for a change.
Researchers at St. Louis University (my alma mater) have developed a treatment for Duchenne muscular dystrophy (DMD), a pernicious wasting disease.
From the SLU website:
"DMD is a fatal form of a muscle wasting disorder that affects one out of every 5,000 to 10,000 boys. The illness is caused by mutations in a gene on the X chromosome. With treatment, those with DMD have an average lifespan of around 25 years. Boys with the illness typically need to use a wheelchair by age 12 and require mechanical ventilation to help with breathing. Many eventually suffer cardiac or respiratory failure.
Thomas Burris, Ph.D., chair of pharmacology and physiology at Saint Louis University and Colin Flaveny, Ph.D., assistant professor of pharmacology and physiology at SLU, study natural hormones that regulate nuclear receptors. By understanding how the body’s natural hormones operate, they aim to develop synthetic compounds to target these receptors in order to create drugs to treat diseases.
In the course of this work, Burris and Flaveny have explored the roles of the nuclear receptor REV-ERB, which regulates key processes in the body, from sleep to cholesterol, and, most recently, muscle regeneration.
"Recently, we found that REV-ERB appears to play unique roles for each stages of muscle tissue development,†Flaveny said.
Muscle stem cells which help replace damaged muscle tissue produce myoblasts that will either reproduce (proliferate) or form muscle tissue (differentiate).
A decline in expression of REV-ERB leads to myoblast differentiation. Conversely, an increase in REV-ERB expression is involved in the regulation of mitochondrial and metabolic function in fully differentiated skeletal muscle.
The team showed that REV-ERB is a regulator of muscle differentiation, and that a drug that inhibits this receptor, called SR8278, stimulates muscle regeneration after acute injury.
End excerpt.
The drug SR8278 has effectively slowed the progression of DMD in animal trials. The researchers are confident it will be effective in humans as well.
This is good news for those suffereing from this horrible illness.
Timothy Birdnow
Some good news for a change.
Researchers at St. Louis University (my alma mater) have developed a treatment for Duchenne muscular dystrophy (DMD), a pernicious wasting disease.
From the SLU website:
"DMD is a fatal form of a muscle wasting disorder that affects one out of every 5,000 to 10,000 boys. The illness is caused by mutations in a gene on the X chromosome. With treatment, those with DMD have an average lifespan of around 25 years. Boys with the illness typically need to use a wheelchair by age 12 and require mechanical ventilation to help with breathing. Many eventually suffer cardiac or respiratory failure.
Thomas Burris, Ph.D., chair of pharmacology and physiology at Saint Louis University and Colin Flaveny, Ph.D., assistant professor of pharmacology and physiology at SLU, study natural hormones that regulate nuclear receptors. By understanding how the body’s natural hormones operate, they aim to develop synthetic compounds to target these receptors in order to create drugs to treat diseases.
In the course of this work, Burris and Flaveny have explored the roles of the nuclear receptor REV-ERB, which regulates key processes in the body, from sleep to cholesterol, and, most recently, muscle regeneration.
"Recently, we found that REV-ERB appears to play unique roles for each stages of muscle tissue development,†Flaveny said.
Muscle stem cells which help replace damaged muscle tissue produce myoblasts that will either reproduce (proliferate) or form muscle tissue (differentiate).
A decline in expression of REV-ERB leads to myoblast differentiation. Conversely, an increase in REV-ERB expression is involved in the regulation of mitochondrial and metabolic function in fully differentiated skeletal muscle.
The team showed that REV-ERB is a regulator of muscle differentiation, and that a drug that inhibits this receptor, called SR8278, stimulates muscle regeneration after acute injury.
End excerpt.
The drug SR8278 has effectively slowed the progression of DMD in animal trials. The researchers are confident it will be effective in humans as well.
This is good news for those suffereing from this horrible illness.
Posted by: Timothy Birdnow at
11:29 AM
| No Comments
| Add Comment
Post contains 344 words, total size 3 kb.
22kb generated in CPU 0.034, elapsed 0.4325 seconds.
35 queries taking 0.4093 seconds, 170 records returned.
Powered by Minx 1.1.6c-pink.
35 queries taking 0.4093 seconds, 170 records returned.
Powered by Minx 1.1.6c-pink.
