Antibiotics against a genetic disease?

18:49
Antibiotics against a genetic disease? -

Some antibiotics have more going for them than their power to kill bacteria. Mouse studies published in the August Journal of Clinical Investigation show that some of these drugs can also be used to treat Duchenne muscular dystrophy, a genetic disease in which victims become paralyzed by progressively weak and generally muscles die in their teens. or in the early 20

A class of antibiotics called aminoglycosides stifle infection closely binding to ribosomes - cellular factories that translate RNA into proteins - invasive bacteria, thus locking them to the low. The same compounds also lock on slightly different ribosomes in animal cells, but not too closely. Consequently, the assembly line of host proteins becomes a little sloppy: Sometimes the wrong amino acid is inserted into a growth protein. The same vagueness can make a ribosome ignore so-called stop codons. These nucleotide sequences normally appear at the end of a gene, where they act as flags, alerting the ribosome protein construction is complete. When a mutation produces a stop codon in the wrong place, it can lead to truncated and therefore worthless protein. Aminoglycosides can watch as the ribosome ignoring the stop sign, allowing him to make the full-length protein.

Researchers have seized this as a possible way to treat Duchenne, in which a truncated version of a protein called dystrophin weakens skeletal muscles. When a team led by physiologist Lee Sweeney of the University of Pennsylvania in Philadelphia gave the aminoglycoside gentamicin to two groups of mice with a disease similar to Duchenne for 2 weeks, animals showed less impairment signs that a control group, and their blood levels of creatine kinase, an enzyme that leaks out of damaged muscle, were 60% lower. Their muscles - after being abducted -. Did not tear as easily and contained more dystrophin

gentamicin can cause serious side effects such as hearing loss or kidney damage, but Sweeney hopes that low doses can be effective and safe, and provides for clinical trials on a small scale. Finally, David Bedwell said microbiologist from the University of Alabama at Birmingham, gene therapy can simply replace the defective gene in patients of Duchenne muscles. But in the meantime, he said, a drug plan "can improve the quality of life and life expectancy of these patients."

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