22 ms·
That´s also my understanding (and my hope). My bet is on heteroplasmic shift though. This will not actually "cure" the disease, but it will minimize its effects
by fasteo 12y ago
That´s also my understanding (and my hope). My bet is on heteroplasmic shift though. This will not actually "cure" the disease, but it will minimize its effects. Specifically, I am closely following the work with mitoTALENs [1]. I have no scientific background, but I find this route easier, both technically and legally. Note the Dr. Aubrey de Grey does not agree with me either [2] :)
[1] Mutation specific compounds that will locate and degrade mutant mitochondria. Once degraded, mutant mitochondria will go through the normal cellular housekeeping (apoptosis) http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html
[2] http://www.reddit.com/r/Futurology/comments/28e4v3/aubrey_de_grey_ama/cia21sf http://www.reddit.com/r/Futurology/comments/28e4v3/aubrey_de...
- exratione 12y agoI agree that mitoTALENs look promising, as does some early work on delivering RNA to mitochondria so that the necessary proteins get made even in the absence of genes. That said, I'd have said the same about exploiting mechanisms in leishmania [1] to deliver useful molecules to mitochondria if you'd asked me five years ago. That line of work seems to be in the doldrums too at this point, not much progress to be seen. The most promising sign is the metasign - that we can compare and contrast so many different approaches, and complain because some are not getting more attention. Yet overall things are happening. [1] http://www.sciencemag.org/content/314/5798/471 http://www.sciencemag.org/content/314/5798/471
- fasteo 12y agoYeah, I feel kind of lucky these days, now that mitochondria seems to be at the center of every possible "aging" disease. Something good will happen sooner or later. The next milestone is the approval of EPI-743[1]. Phase III trials have just finished and data is being gathered and analyzed. If everything works out fine, we could see actual prescriptions of the drug one year from now. Speaking of LHON, take a look at the impressive results[2] of this drug with LHON patients. [1] http://edisonpharma.com/ http://edisonpharma.com/ [2] http://www.ncbi.nlm.nih.gov/pubmed/22410442 http://www.ncbi.nlm.nih.gov/pubmed/22410442