A new study has shown that a single injection of a novel adeno-associated vector (AAV)-based therapy can result in improved enzyme activ-ity and glycogen clearance as well as prolonged survival in a mouse model of Pompe dis-ease. ...
Aug 13, 2018
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Groundbreaking research demonstrates proof-of-concept for using CRISPR-Cas9 genome editing technology to correct the gene mutation responsible for alpha-1 antitrypsin (AAT) deficiency, successfully making a targeted gene ...
Jul 2, 2018
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A new study that compared six of the most promising adeno-associated viral (AAV) gene therapy vectors in human retinal organoid models showed clear distinctions in the efficiency of gene transfer to both retinal pigment epithelial ...
Jun 13, 2018
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During the first 18 months after treatment with ali-pogene tiparvovec, a gene therapy recently approved in Europe to treat lipoprotein lipase deficiency (LPLD), the first patient to receive the treatment had no abdominal ...
May 1, 2018
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Researchers engineered a donor cornea, introducing two genes intended to prevent new blood vessel formation following transplantation, and have shown this novel approach to be safe, well tolerated, and effective at reducing ...
Apr 12, 2018
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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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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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