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Category Archives: Genetic Therapy

CRISPR therapy preserves hearing in genetic deafness model

Hearing loss is the most common form of sensory loss in humans, and almost half of cases have an underlying genetic cause. Reporting today in Nature, a team led by researchers from the Broad Institute of MIT and Harvard, Massachusetts Eye and Ear, Harvard University, and Howard Hughes Medical Institute (HHMI) hasdeveloped a CRISPR-Cas9 genome editing therapy to prevent hearing loss in a mouse model of human genetic progressive deafness. Continue reading

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Gene therapy – About – Mayo Clinic

Overview Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease. Continue reading

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Center for Embryonic Cell and Gene Therapy | Center for …

Science News editors have identified “CRISPR geneediting moves into humans, spurs debate”as their Number 2 story of 2017. OHSU scientists Shoukhrat Mitalipov and Paula Amato’s CRISPRresearch was one of @ScienceNews’ top stories of 2017! #SNTop10 Mitalipov successfully repairs genes in human embryos A ground breaking discovery by Shoukhrat Mitalipov, Ph.D.,was reported in Nature the successful removal of a lethal geneticdefect in human embryos Continue reading

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Gene therapy comes of age | Science

Gene therapy: The power of persistence Nearly 50 years after the concept was first proposed, gene therapy is now considered a promising treatment option for several human diseases. The path to success has been long and tortuous. Serious adverse effects were encountered in early clinical studies, but this fueled basic research that led to safer and more efficient gene transfer vectors Continue reading

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FDA approves gene therapy for blindness – CNN

Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc. The one-time treatment is approved for children and adults with retinal dystrophy due to a mutation of the RPE65 gene, which causes severe visual impairment beginning in infancy. As it progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness. Continue reading

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Gene therapy for rare form of blindness wins U.S. approval …

Matthew Perrone, Associated Press Dr. Albert Maguire, right, helped develop the genetic therapy to treat a type of blindness. Continue reading

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