A new study comparing the effectiveness of individual ZMapp antibodies versus a cocktail of antibodies, administered to mice using recombinant adeno-associated virus (rAAV) delivery vectors, showed the ability to achieve ...
Mar 9, 2018
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Researchers have described severe, life-threatening toxicity in monkeys and piglets given high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector capable of accessing spinal cord neurons. The liver ...
Jan 30, 2018
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Localized gene delivery to diseased joints to achieve sustained drug production at the site of osteoarthritis or rheumatoid arthritis is gaining momentum, with clinical trials underway in the U.S. and the first arthritis ...
Jan 22, 2018
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After three decades of hopes tempered by setbacks, gene therapy—the process of treating a disease by modifying a person's DNA—is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. ...
Jan 11, 2018
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Researchers at Fred Hutchinson Cancer Research Center have identified a specific subset of adult blood stem cells that is exclusively responsible for repopulating the entire blood and immune system after a transplant.
Nov 1, 2017
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Virotherapy capable of destroying tumor cells and activating anti-tumor immune reactions, and the use of engineered hematopoietic stem cells (HSCs) to deliver replacement genes that have the potential to cure blood diseases ...
Oct 18, 2017
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A new study demonstrates success in using a shortened form of the CEP290 gene for gene therapy in a mouse model of Leber congenital amaurosis type 10 (LCA10), a retinal degenerative disorder that causes childhood blindness. ...
Oct 5, 2017
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Researchers added a scaffold/matrix attachment region (S/MAR) to a conventional adeno-associated virus (AAV) vector used for gene transfer, and the modified vectors were able to establish colonies and maintain long-term transgene ...
Sep 22, 2017
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Researchers used gene transfer to block the expression of one of the two main enzymes that break down alcohol in the liver, leading to the accumulation in liver cells of acetaldehyde, a metabolic byproduct of ethanol. The ...
Sep 18, 2017
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A growing body of clinical evidence shows that transplantation of a patient's own mesenchymal stem cells (MSCs) to achieve a cure and prevent recurrent of Crohn's disease-related fistula can be a safe and effective addition ...
Sep 15, 2017
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