5 ms·

Fortunately you probably won't have to go more than another ten years or so with that condition before it can be treated via either allotopic expression [1] of

Fortunately you probably won't have to go more than another ten years or so with that condition before it can be treated via either allotopic expression [1] of mitochondrial genes (e.g. as demonstrated for the single gene mutation in LHON [2]), delivery of whole replacement mitochondria [3], or possibly protofection of an entire replacement mitochondrial genome, although that latter item seems to have stumbled to one of those funding-related halts so common in research [4]. The others are presently pursued, though by no means as aggressively as the potential benefits merit. Everyone need mitochondria repair, not just people with inherited or other global genetic defects of the mitochondria. [1]: https://en.wikipedia.org/wiki/Allotopic_expression https://en.wikipedia.org/wiki/Allotopic_expression [2]: http://archopht.jamanetwork.com/article.aspx?articleid=1814538 http://archopht.jamanetwork.com/article.aspx?articleid=18145... [3]: http://dx.doi.org/10.1016/j.transproceed.2013.11.133 http://dx.doi.org/10.1016/j.transproceed.2013.11.133 [4]: https://www.fightaging.org/archives/2012/10/what-happened-to-protofection.php https://www.fightaging.org/archives/2012/10/what-happened-to...

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 ago
  I 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 ago
    Yeah, 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

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 ago
I 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 ago
  Yeah, 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