A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward ...
Mar 31, 2017
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Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings over the years and are a proven and effective tool that can be used ...
Mar 20, 2017
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Overwhelming evidence from the biomedical literature shows that adeno-associated virus 2 (AAV2), a viral vector often used to deliver therapeutic genes, is not associated with cancer and, in fact, may protect against cancer. ...
Feb 23, 2017
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Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral (AAV) vector. The ability ...
Jan 27, 2017
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Targeting therapeutic genes to the lungs offers the potential to manage serious lung diseases that do not respond to other forms of treatment and to use the lungs as metabolic factories to produce therapeutic proteins for ...
Jan 25, 2017
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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 ...
Dec 30, 2016
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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 ...
Dec 29, 2016
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Investigators from Whitehead Institute, the Ragon Institute of MGH, MIT and Harvard and the Broad Institute of MIT and Harvard have used CRISPR-Cas9 gene-editing technology to identify three promising new targets for treatment ...
Dec 19, 2016
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Gene therapy to repair damaged heart muscle is most likely to succeed if it can be injected at the site of ischemia where there is viable myocardium with reduced contractile ability, and a new technique that combines imaging ...
Nov 15, 2016
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For the first time, National Institutes of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). ...
Oct 26, 2016
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