I think both Ying-Hui and I had the impression that the research had to be somewhat further along before any profit-minded people would fund it. But someone recently explained to me that Silicon Valley companies are often funded with much less scientific backing than this, so this week I’ve written to one venture capitalist I know, and will probably contact others. Regarding getting funding from an existing company, I don’t know much about that option.
Advice is appreciated.
Presumably, it depends a lot on what the gene does. If it affects how your bones grow during childhood, and you’re now an adult, then you’re out of luck. If it produces a protein that has effects in the present, is easy to synthesize and deliver to the body where it matters, then it’s straightforward. (For example, if you want to digest lactose as an adult and lack the right genes, you can buy lactase pills apparently for 10¢.)
I’d also say that the complexity of the phenomenon may be very different from the complexity and difficulty of the cure: e.g. if you had some broken enzyme that caused your body to waste half of some essential vitamin, the consequences to your body might be very complex, but the solution might be to just take a vitamin supplement. At any rate, Wiki says that there are over 6000 genetic disorders, and that over 600 are treatable. The citation on the latter describes a database of known treatments for genetic disorders, and has some interesting numbers:
Regarding the FNSS genes. For DEC2 in particular, it’s the interaction with orexin that makes the difference (the second DEC2 paper showed that an orexin receptor antagonist turned off the short-sleeper effect), and the orexin producers and receptors are all neurons in the brain. And ADRB1, NPSR1, and GRM1 all affect some kind of wakefulness-related receptor (it’s the R in all their names), which I expect are also in the brain. Delivering to the brain is more delicate than to the stomach, but it’s certainly doable.
My impression is that, by trying out different chemicals, you can hope to find one that binds to the receptor and either increases or decreases the activity of it (an “agonist” or “antagonist”) and ideally doesn’t bind to anything else; Ying-Hui said something along those lines. That’s roughly the extent of my knowledge.