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Saturday, December 31, 2016

news-medical : reported that Gene therapy for liver disease advanced to human testing

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daily : 2016-12-31 & on time : 11:1


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AAV-based gene therapy to treat liver disorders advances into human testing


AAV-based gene therapy to treat liver disorders advances into human testing
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Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.The potential for continued technological improvements to expand the therapeutic applications of gene therapy to treat liver disorders and the remaining clinical challenges are examined in a comprehensive review article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.The article is available open access on the Human Gene Therapy website.
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Gene therapy to prevent vision loss: Studies in primates needed to optimize human trials


Gene therapy to prevent vision loss: Studies in primates needed to optimize human trials
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New Rochelle, NY, December 29, 2016--Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying them first in non-human primates before initiating clinical trials, as shown by the results of a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.The article is available free on the Human Gene Therapy website until January 29, 2017.A team of researchers led by Jean Bennett, Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, evaluated and compared two adeno-associated virus (AAV) vectors designed to deliver therapeutic genes directly to the eye when administered via subretinal injection to non-human primates.
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Gene therapy for liver disease advanced to human testing


Gene therapy for liver disease advanced to human testing
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NEW ROCHELLE, N.Y., Dec. 30 (UPI) -- Liver-directed gene therapy using adeno-associated viral, or AAV, vectors to treat diseases like hemophilia have moved on to human testing.The research, conducted by Lisa Kattenhorn and co-authors from Dimension Therapeutics in Cambridge, Mass., show the potential for additional technological improvements to expand the therapeutic applications of gene therapy to treat liver disease.The field of AAV gene therapy has progressed significantly over the past decade with the use of new capsid serotype and organ-specific promoters, scientists have developed an increased understanding of the immune response to AAV in liver-directed therapy.
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