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Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy ...

NOVATO, Calif., May 09, 2022 (GLOBE NEWSWIRE) Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, ....

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Decades after a tragic failure, gene therapy successfully treats a rare liver disease

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Josh McQuillin, shown in 2019, 1 year after being treated, says gene therapy has transformed his life.
© VIT SIMANEK/CTK PHOTO/ALAMY LIVE NEWS
Decades after a tragic failure, gene therapy successfully treats a rare liver disease
May. 18, 2021 , 4:15 PM
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence. ....

United States , Cary Harding , Ada Hamosh , Laurie Zoloth , Jocelyn Kaiser , Josh Mcquillin , James Wilson , Jesse Gelsinger , Paul Gelsinger , Emil Kakkis , Johns Hopkins University , University Of Chicago Divinity School , American Society Of Gene Cell Therapy , Oregon Health Science University , University Of Pennsylvania Upenn , American Society , Cell Therapy , President Emil Kakkis , Oregon Health , Science University , Uchicago Divinity School , ஒன்றுபட்டது மாநிலங்களில் , கேரி கடினப்படுத்துதல் , ஜாஸ்லைந் கைசர் , ஜேம்ஸ் வில்சன் , ஜெஸ்ஸி கெள்சிங்கேர் ,