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#gene therapy

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Gene Therapy Advances To Better Treat ‘Bubble Boy’ Disease

Author: Richard Harris / Source: NPR.org Sometimes rare diseases can let scientists pioneer bold new ideas. That’s been the case with a condition that strikes fewer than 100 babies a year in the United States. These infants are born without a functioning immune system. The disease is called
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FDA Approves First Ever Gene Therapy Treatment For Blindness

Author: Good News Network / Source: Good News Network 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 FDA’s blessing makes Luxturna, a
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CRISPR/Cas9 can reverse multiple diseases in mice

Author: Tina Hesman Saey / Source: Science News PUMP IT UP A new twist on gene editing makes the CRISPR/Cas9 molecular scissors act as a highlighter for the genetic instruction book. Such highlighting helps turn on specific genes. Using the new tool, researchers treated mouse versions of type 1
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Gene Therapy Gives a Boy with Epidermolysis Bullosa His Life Back

Author: Robby Berman / Source: Big Think Among the most promising advances in medicine recently has been developing the ability to “heal” misbehaving genes by replacing defective elements with healthy ones through “gene therapy.” A recent case offers some startling evidence of how effective this
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Scientists replaced 80 percent of a ‘butterfly’ boy’s skin

Author: Tina Hesman Saey / Source: Science News In a last-ditch effort to save a dying 7-year-old boy, scientists have used stem cells and gene therapy to replace about 80 percent of his skin. This procedure’s success demonstrates that the combination therapy may be effective against some rare
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