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Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes-called meganucleases-with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells.
Read more: Repair Possible of Gene Responsible for Duchenne Muscular Dystrophy http://www.medindia.net/news/Repair-Possible-of-Gene-Responsible-fo...
So what's we've learnt this year is that MDX is not a reliable animal model... Good results in MDX do not mean much unfortunately. We'll see what happens with ACE-031...I did not see any data other than MDX and post-menoupausal women on that one. Sure hope that they have more data...
As for urtrophin upregulation, PTC has a compund that is "planned" to enter clinics in 2011. Not sure if PTC has resources to work on this before they figure out what to do with Ataluren though.
I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said:So what's we've learnt this year is that MDX is not a reliable animal model... Good results in MDX do not mean much unfortunately. We'll see what happens with ACE-031...I did not see any data other than MDX and post-menoupausal women on that one. Sure hope that they have more data...
As for urtrophin upregulation, PTC has a compund that is "planned" to enter clinics in 2011. Not sure if PTC has resources to work on this before they figure out what to do with Ataluren though.
I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said:So what's we've learnt this year is that MDX is not a reliable animal model... Good results in MDX do not mean much unfortunately. We'll see what happens with ACE-031...I did not see any data other than MDX and post-menoupausal women on that one. Sure hope that they have more data...
As for urtrophin upregulation, PTC has a compund that is "planned" to enter clinics in 2011. Not sure if PTC has resources to work on this before they figure out what to do with Ataluren though.
In the studies for how long do they do the tests on MDX before taking to clinical trials. Do they tried the Ace 31 with kids with duchenne.
Jeff Sobel said:I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said:So what's we've learnt this year is that MDX is not a reliable animal model... Good results in MDX do not mean much unfortunately. We'll see what happens with ACE-031...I did not see any data other than MDX and post-menoupausal women on that one. Sure hope that they have more data...
As for urtrophin upregulation, PTC has a compund that is "planned" to enter clinics in 2011. Not sure if PTC has resources to work on this before they figure out what to do with Ataluren though.
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