Gene therapy may be the best hope for curing retinitis pigmentosa (RP), an inherited condition that usually leads to severe vision loss and blinds 1.5 million people worldwide.
Mar 21, 2024
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A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of ...
Jan 30, 2024
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Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman ...
Oct 10, 2022
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A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop ...
Apr 17, 2019
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The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie "Lorenzo's Oil."
Oct 4, 2017
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Scientists at The Scripps Research Institute have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating ...
Jul 1, 2012
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Up to half of all prostate cancer cells have a chromosomal rearrangement that results in a new "fusion" gene and formation of its unique protein -- but no one has known how that alteration promotes cancer growth. Now, Weill ...
May 22, 2012
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Their stories are divided into before and after. First, those long years of pain which flooded every moment—school, relationships, work.
Mar 18, 2024
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Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the enzyme UGT1A1 which is responsible for metabolizing it into a substance that ...
Aug 17, 2023
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An experimental gene therapy that's applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease.
Dec 15, 2022
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