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Researchers Find Genetic Cause For Alzheimer's, Possible Method To Reverse It (upi.com)
schwit1 quotes UPI: Scientists at an independent biomedical research institution have reported a monumental breakthrough: The cause of the primary genetic risk factor for Alzheimer's disease, and a possible cure for the disease. Researchers at Gladstone Institutes in San Francisco identified the primary genetic risk factor for the disease, a gene called apoE4... Their findings were published this week in the journal Nature Medicine... By treating human apoE4 neurons with a structure corrector, it eliminated the signs of Alzheimer's disease, restored normal function to the cells and improved cell survival.
The study's senior investigator says he's already working with a San Francisco pharmaceutical startup to develop the approach and move towards clinical trials, adding that "we are working to accelerate the timeline as much as possible."
The study's senior investigator says he's already working with a San Francisco pharmaceutical startup to develop the approach and move towards clinical trials, adding that "we are working to accelerate the timeline as much as possible."
I've lost two close family members to Alzheimers. It's one of the cruelest diseases I've ever seen. Yes, we want a damn cure.
"Tell me doctor, with all of your defenses, are there any provisions for an attack by killer bees?"
Part of the importance is that they use a "small molecule inhibitor" (SMI). This class of medical tins are usually easy to manufacture and ingest. In contrast, most of the other "breakthrough"molecules ave been genetically engineered proteins which are more complex. In addition, SMI drugs can be easily modified to have better profiles.
You're correct. The paper is about a cell culture model for Alzheimer's. The authors point out that lots of potential treatments seem to work on mouse models but fail miserably in humans. So they created a cell culture model using stem-cell derived human neurons. They show that neurons that express ApoE4 have various Alzheimer's-like features, and that these can be reversed by gene editing to flip the ApoE4 to another variant, or through the use of a structure-correcting drug.
The paper is really about the cell culture model, which is very important, but it's not a new drug, and it's a long way from being an actual human.