Gene therapy holds promise for treating
inherited Lou Gehrig's Disease
eurekalert.org
The GDNF gene therapy enhanced survival of motor neurons in the laboratory model of inherited ALS and increased the number of motor neurons that maintained 'communication' with cells in treated leg muscles. Additionally, GDNF gene therapy slowed shrinkage of motor neurons and muscle atrophy, improved motor function in affected mice and delayed onset of ALS disease symptoms.
"The most critical issues in designing a gene therapy approach for any human disease are to have a good animal model of the disease and to choose the appropriate target tissue and mode of gene delivery," Bohn said.
The mice used in this study express a mutant gene that is carried by humans with a hereditary form of ALS. These mice were developed by Mark Gurney and Teepu Siddique, professor of neurology at Northwestern University Medical School.
I thought you might like to hear that bit about having a good animal
model Martin--I hope that the Ariad mice in that PNAS paper bring the
stock a little attention--but it is also possible that people have
heard so often, for example, the ease with which tumors are cured in
mice, that mice just dont mean anything to the public anymore--and
it is not as if Ariad brought attention to the paper with a PR. The
Ariad gene therapy paper will not result in the stock moving much,
if at all--yup, down 1/32 as I type this.
The genetically modified ARGENT mice are going to be running
around inside their wheels, with regulated EPO, insulin, and
higher bone density--and we will still be over here spinning
our wheels.
Well, I'm sick to death of this--logging off for a few
days. Maybe Ariad will move if we don't watch the stock.
And if my stocks didn't do anything for the rest of the
year I'd still have a very good percentage gain...(click). |
