An experimental gene therapy developed by UCLA, University College London, and Great Ormond Street Hospital has successfully restored immune function in 59 of 62 children born with ADA-SCID, a rare…
GENE THERAPY
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A single-dose gene therapy has demonstrated remarkable results in slowing the advancement of Huntington’s disease during a preliminary trial, according to a press release from University College London (UCL). Researchers…
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The Long-Awaited Three-Year Study Results on Huntington’s Disease AMT-130, uniQure’s gene therapy, would be the first genetic treatment for Huntington’s disease, if approved. The observed slowing of disease progression is…
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The following review from Gene Therapy of Nature presents the advances and future prospects of gene editing in relation to collagen disorders. Collagen disorders include a broad spectrum of inherited…
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A research group funded by the National Institutes of Health (NIH) has successfully created and delivered a personalized gene-editing therapy to treat an infant suffering from a life-threatening, previously untreatable…
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A large research group led by Dr. Jeff Vierstra, a researcher at the Altius Institute for Biomedical Sciences in Seattle (USA), and Dr. Nikoletta Psatha, Assistant Professor in the Department…
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In a major medical breakthrough, India has successfully completed its first human gene therapy for severe hemophilia-A using a lentiviral vector, resulting in zero bleeding rates in patients. Hemophilia, a…
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Scientists at Trinity College Dublin have developed a very promising gene therapy to treat glaucoma, a debilitating eye condition that affects around 80 million people globally and can result in…
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Once a death sentence, hemophilia may soon have a treatment. A hereditary condition that primarily affects boys is caused by blood clotting failure. After an injury, hemophilia can result in…
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Children’s Hospital of Philadelphia (USA) corrected genetic hearing loss using a gene therapy procedure developed in Italy. An 11-year-old kid with a rare type of congenital deafness has been able…
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