A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up ...
Aug 15, 2018
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Researchers at UMass Medical School have developed a genome-editing strategy to correct disease-causing DNA mutations in mouse models of human genetic diseases, according to research published in the Aug. 18 edition of Nature ...
Aug 14, 2018
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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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OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people—a finding that holds promise for developing new gene therapies to treat Batten ...
Jul 27, 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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Parents cherish developmental milestones, from a newborn's grip of an offered finger; to an infant's holding her head up the first time; to rolling over, creeping, and crawling; then to standing, cruising, and finally walking. ...
Jun 8, 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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