The Longecity community is presently raising funds for a modestly-sized rejuvenation research project to be undertaken by a SENS Research Foundation team. Grand goals are made of many small steps, and these days any given small step in the life sciences can take the form of a six month project, a few skilled post-graduate researchers with access to an established lab, and $10-30,000. Meaningful research that pushes forward the boundaries of medical science is becoming very cheap, with that fall in prices driven by accelerating progress in the tools and capabilities of biotechnology. We can see this process at work here, as this significant and useful gene therapy research can be conducted with a proposed budget of $21,000.
In this project, engineered mitochondrial genes will be used to restore function to cells that contain defective mitochondrial genes.
The SENS team is developing a unique method for targeting these genes to the mitochondria; this step has been the bottleneck in research on this topic over the last decade. In their system, the mRNA from the engineered mitochondrial gene is targeted to the mitochondrial surface before it is translated into a protein using a co-translation import strategy. Once imported, it is incorporated into the correct location in the inner mitochondrial membrane. The long-term goal of this project is to utilize this improved targeting strategy to rescue mutated mitochondrial DNA and thereby prevent and cure one of the major causes of cellular aging.
There is an open question and answer thread dedicated to the project in the Longecity forums, and the researchers have started to discuss the research in some detail. I think that you'll find it interesting:
One reason [that we chose to work with the genes CyB and ATP8] that these may be both the easiest and hardest genes to achieve efficient import with. CyB has a reputation (whether or not it is deserved is a matter for some debate) in the field of being the most difficult and hydrophobic protein to import into the mitochondria. It is one of the bigger mitochondrially encoded genes, so at the very least it is a challenge. ATP8, on the other hand, is tiny and so may be considered the easiest to import. Thus we've set ourselves a task that spans the range of challenges that we think we'll encounter.
The second reason is that, strategically, OxPhos complexes III and V are the most interesting for proof of concept rescue of the entire mito genome. The reason is that they have the fewest genes that are encoded by the mitochondria. Complex III has only CyB (and thus ONLY CyB is needed to rescue the entire complex) and Complex V has only 2: ATP6 and ATP8. So if we want to study functional rescue of entire complexes then III and V are the easiest.
A third of the needed project funds - $7,000 - will be raised from the community while the remaining $14,000 will matched by Longecity: $2 for every $1 donated. Since I last mentioned this project, donors have contributed a little over $5,000 - so just $2,000 left to go, with a November deadline. If you've been on the fence about donating, then jump in! It's never too late to help build a future that we'd all like to live in, one that involves far longer, healthier lives, and complete prevention of the diseases of aging.
This mitochondrial gene therapy project is exactly the sort of crowdfunded science initiative that I like to see succeed - and that I'd like to see succeed again many times over in the years ahead. A future in which the SENS Research Foundation obtains a significant amount of its science budget from the community on a project by project basis is a bright one, I think. It isn't just a matter of money and connecting with supporters: it also generates publicity, materials, and broader interest in the research itself. At this stage in the game it is still very important to talk to the rest of the world about longevity science and the prospects for progress in the near future, and crowdfunding rejuvenation research via Longecity, Microryza, and Indiegogo - and their successors - blends fundraising, organizational transparency, advocacy, education, and persuasion in a very useful way.