The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV). The study, which showed that this strategy enabled gene ...
Mar 22, 2023
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While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps. A new study published in Blood Advances finds that the average ...
Mar 16, 2023
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A new research paper titled "AAV1.NT-3 gene therapy prevents age-related sarcopenia" has been published in Aging.
Mar 15, 2023
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People with sickle cell disease who receive nonmyeloablative stem cell transplants are at greater risk for developing new tumors, according to a new study published in the Journal of Clinical Oncology.
Mar 9, 2023
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Pancreatic cancer is an incurable form of cancer, and gene therapies are currently in clinical testing to treat this deadly disease. A comprehensive review of the gene and cell biotherapies in development to combat pancreatic ...
Mar 6, 2023
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For patients with hemophilia B, one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec) is noninferior and superior to prophylaxis with factor IX, according ...
Mar 3, 2023
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Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in Human Gene Therapy.
Feb 22, 2023
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Could humanity finally be gaining the upper hand in our age-old fight against cancer?
Feb 2, 2023
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A gene therapeutic approach that shows promise for treating age-related macular degeneration (AMD) may also be effective in treating other eye conditions such as glaucoma. It provided benefit in three additional models of ...
Jan 30, 2023
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Researchers working with UMass Chan Medical School's Translational Institute for Molecular Therapeutics announced progress in developing a vector to deliver gene replacement therapy in mice models with Cockayne syndrome, ...
Jan 30, 2023
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