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Posted On: 02/05/2022 5:57:42 PM
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Major Coverage of Wolfram Syndrome in Science
The below portion is referring to MANF Gene Therapy
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. ..... The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.....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.
https://www.science.org/content/article/race-...RN.twitter
The below portion is referring to MANF Gene Therapy
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. ..... The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.....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.
https://www.science.org/content/article/race-...RN.twitter
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