News-Medical.Net Gene Therapy News
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Virally mediated gene therapy could potentially treat genetic hearing loss
May 27, 2023 | 04:40 amBy 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic hearing loss is often the most difficult to treat.
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Nonhuman primate study helps explain how stem cell transplantation can kill HIV virus
May 25, 2023 | 20:50 pmNew animal research is helping explain why at least five people have become HIV-free after receiving a stem cell transplant.
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Bridging the Gap between Academia and Industry: The Power of Collaboration in Life Sciences
May 25, 2023 | 08:48 amIn this behind-the-scenes interview, we speak to Dr. Sinead Knight, chair of SLAS Europe 2023, about this years programme, highlighting the key topics and speakers.
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Study provides data to support gene supplementation as a treatment for autosomal dominant retinitis pigmentosa
May 23, 2023 | 20:59 pmA new study shows that total RPE65 protein levels in mice with autosomal dominant retinitis pigmentosa were doubled following subretinal delivery of adeno-associated virus (AAV)-RPE65 gene supplementation.
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MGH researchers identify a potential method for treating fragile X syndrome
May 19, 2023 | 22:46 pmNew research has identified a potential method for treating fragile X syndrome, a leading cause of autism spectrum disorders that is characterized by an inherited repeat of certain nucleotides within the DNA sequence of the FMR1 gene.
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New technique could mechanically enhance glymphatic transport in the mouse brain
May 17, 2023 | 04:19 amLike the lymphatic system in the body, the glymphatic system in the brain clears metabolic waste and distributes nutrients and other important compounds.
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Nemours Children's Health selected to conduct first gene therapy clinical trial for Morquio A syndrome
May 17, 2023 | 02:57 amAs part of the Foundation for the National Institutes of Health Accelerating Medicines Partnership Bespoke Gene Therapy Consortium, Nemours Children's Health has been chosen to conduct a first-of-its-kind gene therapy clinical trial for Morquio A syndrome.
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Phase 1 clinical trial of CAR-NKT cell therapy shows promising results in neuroblastoma patients
May 15, 2023 | 19:24 pmResearchers at Baylor College of Medicine/Texas Children's Cancer Center and collaborating institutions report interim results from a first-in-human phase 1 clinical trial evaluating the safety, antitumor activity and immunological characteristics of a genetically engineered natural killer T (NKT) cell immunotherapy for neuroblastoma, a childhood tumor that most commonly arises in the adrenal gland.
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Patient-derived head and neck cancer organoids could aid the discovery of novel therapies
May 12, 2023 | 19:55 pmResearchers from the Organoid group (Hubrecht Institute) and UMC Utrecht have developed a biobank with organoids derived from patients with head and neck cancer (HNC).
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Cellular Origins partners with ScaleReady to simplify, standardise, and automate cell therapy manufacturing
May 11, 2023 | 15:08 pmCellular Origins a TTP Company focused on enabling scalable, cost-effective, and efficient manufacture of cell and gene therapies (CGTs).
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