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Dr. Urano featured in Science
https://www.sciencemag.org/news/2021/02/race-...s-diabetes
But Urano knows that even if a treatment receives approval, it would be only “a Band-Aid for Wolfram syndrome.” Hoping to develop a genetic cure, he and colleagues are introducing replacement genes into cells from patients and from mice engineered to replicate the disease. The researchers are endowing the cells with healthy copies of the gene for wolframin or the gene for a protein that reduces ER stress to determine whether they restore cellular function and reduce cell death. At INSERM, Delettre and colleagues are also evaluating whether directing a working gene into optic nerve cells can curtail vision loss in mice with faulty wolframin. The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.
Urano agrees, predicting that CRISPR-based Wolfram therapies might take 10 to 20 years to develop. The alternative approach, gene therapy, could reach clinical trials more quickly, in 3 to 10 years, he says, because researchers have more experience with gene therapy and have created several treatments that have already been approved for other illnesses.
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Because it stems from a single genetic glitch, Wolfram syndrome could also help scientists tease out the role of the ER in more complex diseases, including neurological conditions, type 2 diabetes, and cancer. The ER also falters in those diseases, causing cells to die, but the mechanism is harder to discern because they stem from myriad genetic and environmental factors. In Alzheimer’s disease, for instance, neurons develop ER stress as misfolded proteins accumulate inside and outside the cells.
Besides deepening researchers’ understanding of other conditions, the research on Wolfram syndrome might even deliver candidate treatments. “Everyone would be very excited if we can make advances in targeting ER stress in Wolfram syndrome,” Oakes says. “It would open up the whole field to doing this in other degenerative diseases.”
https://www.sciencemag.org/news/2021/02/race-...s-diabetes
But Urano knows that even if a treatment receives approval, it would be only “a Band-Aid for Wolfram syndrome.” Hoping to develop a genetic cure, he and colleagues are introducing replacement genes into cells from patients and from mice engineered to replicate the disease. The researchers are endowing the cells with healthy copies of the gene for wolframin or the gene for a protein that reduces ER stress to determine whether they restore cellular function and reduce cell death. At INSERM, Delettre and colleagues are also evaluating whether directing a working gene into optic nerve cells can curtail vision loss in mice with faulty wolframin. The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.
Urano agrees, predicting that CRISPR-based Wolfram therapies might take 10 to 20 years to develop. The alternative approach, gene therapy, could reach clinical trials more quickly, in 3 to 10 years, he says, because researchers have more experience with gene therapy and have created several treatments that have already been approved for other illnesses.
....
Because it stems from a single genetic glitch, Wolfram syndrome could also help scientists tease out the role of the ER in more complex diseases, including neurological conditions, type 2 diabetes, and cancer. The ER also falters in those diseases, causing cells to die, but the mechanism is harder to discern because they stem from myriad genetic and environmental factors. In Alzheimer’s disease, for instance, neurons develop ER stress as misfolded proteins accumulate inside and outside the cells.
Besides deepening researchers’ understanding of other conditions, the research on Wolfram syndrome might even deliver candidate treatments. “Everyone would be very excited if we can make advances in targeting ER stress in Wolfram syndrome,” Oakes says. “It would open up the whole field to doing this in other degenerative diseases.”
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