New initiatives by the National Center for Advancing Translational Sciences (NCATS) to use gene therapy approaches to treat rare diseases and especially promising aspects of gene transfer and gene editing technology, such ...
Feb 24, 2016
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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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Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with reduced ejection fraction, ...
Jun 6, 2017
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Study results of CD19-directed chimeric antigen receptor (CAR) therapy using the Sleeping Beauty non-viral transduction system to modify T cells has demonstrated further promise in patients with advanced hematologic malignancies.
Dec 8, 2014
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A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, ...
Sep 29, 2015
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Scientists at the Academy of Finland's Centre of Excellence in Computational Inference Research have developed novel computational methods that have yielded essential knowledge of how hospital-acquired bacteria spread and ...
Jan 16, 2013
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Mounting evidence suggests that the secret to understanding human health and combating metabolic diseases lies hidden within the microscopic world of our gut bacteria.
Feb 20, 2024
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Stent angioplasty saves lives, but there often are side effects and complications related to the procedure, such as arterial restenosis and thrombosis. In the June 2013 issue of The FASEB Journal, however, scientists report ...
May 30, 2013
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Nine of twelve leukemia patients who received infusions of their own T cells after the cells had been genetically engineered to attack the patients' tumors responded to the therapy, which was pioneered by scientists in the ...
Dec 10, 2012
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New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events ...
Jul 27, 2016
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