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Category Archives: Genetic Therapy

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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FDA approves gene therapy to fix mutations that can lead …

In a decision that accelerates a new era for gene therapy, the Food and Drug Administration on Tuesday approved a DNA-altering medication that can reverse an inherited form of progressive blindness. The FDAs blessing makes Luxturna, a treatment for retinal dystrophy caused by a specific genetic mutation, the first gene therapy cleared for use in the U.S. to treat a genetic disease other than cancer 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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Ultragenyx Announces FDA Approval of MEPSEVII …

NOVATO, Calif., Nov. 15, 2017 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. Continue reading

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Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage …

Few gene-therapy companies have the factories or expertise to make the viruses for use in clinical trials, where standards are exacting and comprehensive. Continue reading

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