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AAV5 Vector Hemophilia

This post categorized under Vector and posted on June 20th, 2019.
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AAV5 Vector Hemophilia This post categorized under Vector and posted on May 20th 2018. This study evaluates how safe gene therapy treatment with AAV5-hFIX is in graphic patients with severe or moderately severe hemophilia B and severe bleeding type.Hemophilia B. uniQure is advancing a promising clinical program focused on hemophilia B a severe orphan blood clotting disorder.01.03.2018 AAV5 liver-directed wild-type hFIX gene transfer was well tolerated and clinically effective in severe and moderate-severe hemophilia B. No cellular immune responses to the AAV5 vector were detected and FIX expression levels were stable for the entire observation periodCited by 31Publish Year 2017Author Wolfgang Miesbach Karina Meijer Michiel Coppens Peter Kampmann Robert Klamroth Roger SchutgensAMT-061 consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure holds multiple issued patents in the United States and Canada broadly with patent pending in the EU covering methods of treating bleeding disorders including hemophilia B using AAV gene therapy with the FIX-Padua variant.

01.03.2018 In hemophilia B gene therapy either wild-type or Padua factor IX with a liver-specific promoter is inserted into the genome of the AAV vector and administered via a peripheral vein. The vector transduces hepatocytes leading to the production of FIX. The levels of FIX in patients and unaffected individuals before and after gene therapy are shown below. Professional illustration by Author Michael MakrisPublish Year 2018


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