(Medical Xpress)—Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, ...
Dec 9, 2013
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A fusion protein engineered by researchers at KU Leuven combining proteins active in HIV and Moloney murine leukaemia virus (MLV) replication may lead to safer, more effective retroviral gene therapy.
Nov 13, 2013
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Two Houston researchers from Baylor College of Medicine and Texas Children's Hospital were part of an international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited ...
Jul 11, 2013
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A single session of a gene therapy developed by the Universitat Autonoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every ...
Jul 2, 2013
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European scientists have made a major breakthrough in the treatment of inherited retinal diseases.
Jun 20, 2013
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In fall 2012, the European Medicines Agency (EMA) approved the modified adeno-associated virus AAV-LPL S447X as the first ever gene therapy for clinical use in the Western world. uniQure, a Dutch biotech company, had developed ...
Jun 19, 2013
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(Medical Xpress)—A fresh round of trials to evaluate gene therapy for the heart is set to begin in a couple of weeks. The British Heart Foundation will be sponsoring the study, which seeks to replace defective calcium pump ...
Stem cells and tissue-specific cells can be grown in abundance from mature mammalian cells simply by blocking a certain membrane protein, according to scientists at the University of Pittsburgh School of Medicine and the ...
Apr 17, 2013
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Researchers from Basque Public University are using nanotechnology to develop new formulations that can be applied to drugs and gene therapy. Specifically, they are using nanoparticles to design systems for delivering genes ...
Feb 28, 2013
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Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal ...
Dec 19, 2012
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