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Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss

December 20, 2017 –  The American Food & Drug Administration (FDA) has just approved  a novel gene therapy to treat patients with a rare form of inherited vision loss. Voretigene Neparvovec-Rzyl (Luxturna) is the first directly administered gene therapy approved to target a disease caused by mutations in a specific human gene (i.e.,

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  • Scientists use gene-editing tool CRISPR in attempt to cure HIV September 13, 2019
    The first attempt to use the gene-editing tool CRISPR to cure HIV infection was unsuccessful but the approach does show promise.
  • New cardiac fibrosis study identifies key proteins that translate into heart disease September 12, 2019
    Using cutting-edge technologies, researchers at Duke-NUS Medical School, Singapore, have developed the first genome-wide dataset on protein translation during fibroblast activation, revealing a network of RNA-binding proteins (RBPs) that play a key role in the formation of disease-causing fibrous tissue in the heart. Their findings, published in the journal Circulation, could help in the search […]
  • How your genes affect the number on your scale September 12, 2019
    Could your genes be keeping you from losing weight?
  • Scientists identify gene as master regulator in schizophrenia September 11, 2019
    Using computational tools to investigate gene transcription networks in large collections of brain tissues, a scientific team has identified a gene that acts as a master regulator of schizophrenia during early human brain development. The findings may lay the groundwork for future treatments for the highly complex neuropsychiatric disorder.
  • How your DNA takes shape makes a big difference in your health September 11, 2019
    The more we learn about our genome, the more mysteries arise. For example, how can people with the same disease-causing mutation have different disease progression and symptoms? And despite the fact that it's been more than 15 years since the human genome was sequenced, why can't we explain the significance of the vast majority of […]
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