Need to read something super positive and optimistic today? I strongly recommend this new study in press at MCP that totally made my day!
It's really easy to look at the broad range of different genetic mutations that can lead to a single disease and think.....
Retinal degeneration diseases ABSOLUTELY fall in this category. Check out this associated paper on progressive vision loss in dogs.
Mutations on 17 different stupid genes are known to lead to just progressive retinal atrophy - which is just one of many diseases that cause dogs to go blind later in life.
If you are in drug development in either primary research or for applied for-profit stuff what do the odds of success sound like for a disease caused by at least 17 different things? Can you convince someone to help fund you while you chase targets that may only help a small percentage of those afflicted?
Almost always? No. That's a bad elevator pitch and a worse grant application. In pharma? Start sending out CVs before you ask.
Why this paper is so very very cool is that they took some of the mouse models for progressive retinal degradation (mutations on different genes!) and looked at the proteins that actually change vs controls. They're the same!
Unnecessary reminder for most people here (good for outsiders, who still can't seem to get this stuff straight)
Genome is genotype, that's what the DNA says, but that isn't what is physically happening
Proteome is often the phenotype (what is physically happening!) (or at least very close and involved in the phenotype)
AND - Nearly all drugs target proteins!
These authors don't miss the point here either. Who cares what the gene is that caused the protein change if you know the protein causing the problem? Not me, not these authors, and certainly not patients. Cause now you've got something to develop a drug against!
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