Scientists at the Howard Hughes Medical Institute's Janelia Research Campus and the University of California, Berkeley have developed a powerful new tool for neuroscientists—a viral vector called rAAV2-retro, which efficiently ...
Oct 7, 2016
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New initiatives by the National Center for Advancing Translational Sciences (NCATS) to use gene therapy approaches to treat rare diseases and especially promising aspects of gene transfer and gene editing technology, such ...
Feb 24, 2016
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Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual ...
Dec 6, 2015
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Three years ago, a team from the University of Pennsylvania announced that they had cured X-linked retinitis pigmentosa, a blinding retinal disease, in dogs. Now they've shown that they can cure the canine disease over the ...
Oct 12, 2015
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National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping ...
Jan 20, 2015
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A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening condition also known as "bubble boy" disease, appears to be both effective and safe, according to a ...
Oct 8, 2014
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Researchers have hijacked a defense system normally used by bacteria to fend off viral infections and redirected it against the human papillomavirus (HPV), the virus that causes cervical, head and neck, and other cancers.
Aug 9, 2014
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Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome.
Jun 26, 2014
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The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy ...
Apr 6, 2014
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Working in mice, researchers at Washington University School of Medicine in St. Louis report developing a gene delivery method long sought in the field of gene therapy: a deactivated virus carrying a gene of interest that ...
Dec 23, 2013
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