News-Medical.Net Gene Therapy News
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Discovery suggests a new way to treat spinal muscular atrophy
Mar 23, 2023 | 20:18 pmColumbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades.
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Disruption of the FcRn–IgG interaction may make AAV-based gene therapies effective
Mar 22, 2023 | 15:17 pmThe use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV).
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Snapping at new biotech breakthroughs with Gator Pro
Mar 21, 2023 | 13:17 pmIn this interview, we speak to Subodh Nimkar, Marketing Director at Gator Bio, about their latest technology, Gator Pro which allows for fast, reliable, and high-quality data.
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Study highlights a persistent life expectancy gap among patients living with sickle cell disease
Mar 16, 2023 | 15:17 pmWhile research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps.
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Study provides strong support for AAV.NT-3 gene therapy in the management of sarcopenia
Mar 16, 2023 | 09:47 amA new research paper was published on the cover of Aging (listed by MEDLINE/PubMed as "Aging (Albany NY)" and "Aging-US" by Web of Science) Volume 15, Issue 5, entitled, "AAV1.NT-3 gene therapy prevents age-related sarcopenia."
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University of Utah and Tikkunlev partner to bring heart failure gene therapy innovation to the clinic
Mar 8, 2023 | 18:33 pmThe University of Utah, one of the nation's leading research universities, and TikkunLev Therapeutics, a gene therapy company, have announced a new partnership to accelerate an innovative heart-failure gene therapy.
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Common scientific method to analyze DNA in early human embryos fails to accurately reflect gene edits
Mar 7, 2023 | 16:17 pmA commonly used scientific method to analyze a tiny amount of DNA in early human embryos fails to accurately reflect gene edits, according to new research led by scientists at Oregon Health & Science University.
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Fighting pancreatic cancer with gene and cell biotherapies
Mar 6, 2023 | 18:07 pmPancreatic 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 cancer is published in the peer-reviewed journal Human Gene Therapy.
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New delivery system based on lipid nanoparticles could increase the efficiency of gene therapy
Mar 2, 2023 | 16:22 pmLipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.
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Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities
Feb 28, 2023 | 06:26 amA new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.
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