Cancer Research UK scientists are the first to use an efficient new screening strategy to identify gene faults in tumour cells that are possible drug targets for the most common form of lung cancer, according to new research ...
Jul 9, 2013
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Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from ...
Jun 4, 2013
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Using a novel method of analyzing genetic variations in families, researchers at Johns Hopkins have found that individually harmless genetic variations affecting related biochemical processes may team up to increase the risk ...
May 28, 2013
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University of Massachusetts Amherst researchers, experts in revealing molecular structure by X-ray crystallography, have identified two new small "chaperone" molecules that may be useful in treating the inherited metabolic ...
Oct 9, 2012
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Researchers have demonstrated that a refined gene therapy approach safely restores the immune systems of some children with severe combined immunodeficiency (SCID). The rare condition blocks the normal development of a newborn's ...
Sep 11, 2012
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Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated ...
Dec 11, 2011
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An international team led by researchers at UCL and the NIHR Great Ormond Street Hospital Biomedical Research Centre have developed in mice a gene therapy that significantly reduces the hearing loss associated with Norrie ...
Aug 29, 2023
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A 19-year-old man from China, who has been having memory problems since the age of 17, was diagnosed with dementia, according to a recent case study published in the Journal of Alzheimer's Disease.
Feb 23, 2023
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In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short ...
Nov 21, 2022
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Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
Aug 3, 2021
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