Packaging replacement genes in viruses is an effective method to deliver them to target tissues, but the human body mounts an immune response against the virus. The systemic and local immune reactions induced by an adeno-associated ...
Jan 8, 2014
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Recent evidence demonstrating the feasibility of using novel CRISPR/Cas9 gene editing technology to make targeted changes in the DNA of human embryos is forcing researchers, clinicians, and ethicists to revisit the highly ...
May 1, 2015
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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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An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease ...
Aug 13, 2019
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Researchers with the VA San Diego Healthcare System and the University of California San Diego have shown that a gene transfer technique can combat heart dysfunction caused by diabetes.
Mar 27, 2019
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Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival ...
Aug 11, 2020
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Researchers at KU Leuven in Belgium have developed a technique to make sheep produce new antibodies simply by injecting the DNA building blocks. This approach is much cheaper and more efficient than producing antibodies industrially ...
Sep 11, 2019
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Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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A new study has definitively shown that a single treatment with gene therapy using adeno-associated viral (AAV) vector gene delivery to replace the defective gene responsible for congenital adrenal hyperplasia (CAH) will ...
Mar 29, 2018
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Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem ...
Apr 18, 2019
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