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Awesome medical advance

Discussion in 'Environmental Discussion' started by bwilson4web, Dec 8, 2023.

  1. bwilson4web

    bwilson4web BMW i3 and Model 3

    Nov 25, 2005
    Huntsville AL
    2017 Prius Prime
    Prime Plus
    Source: US regulators approve two gene therapies for sickle cell disease | US news | The Guardian

    The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients.

    The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics. Both therapies are made from the patients’ own blood stem cells and were approved for people aged 12 and older.

    The Vertex/Crispr gene therapy uses the breakthrough gene-editing technology that won its inventors the Nobel prize in 2020. The therapy can be directed to cut DNA in targeted areas, enabling the ability to accurately remove, add or replace DNA where it was cut.

    The modified blood stem cells are then transplanted back into the patient, where they attach and multiply within the bone marrow and increase the production of fetal hemoglobin, a type of hemoglobin that facilitates oxygen delivery.

    Lyfgenia is a cell-based gene therapy that modifies a patient’s blood stem cells to produce a gene therapy-derived hemoglobin that functions similarly to a type of normal adult hemoglobin not affected by sickle cell disease.
    . . .

    Some early gene therapy attempts did not go well. But we are a clever species (somedays) and this is awesome.

    Bob Wilson
  2. PriusCamper

    PriusCamper Senior Member

    Mar 3, 2012
    Pacific Northwest, USA
    2007 Prius
    I've been following this one. It's still highly experimental, but promising.

    And of course criminal profiteering of hospital executives, insurance companies, RX industry and others means that if they can't make a killing in profits with this treatment, they deny coverage for the treatment.

    Also, most people don't know it but sickle-cell anemia is NOT more common in black people. That's a racist interpretation that has been clarified with data recently. Of course black people will go longer without diagnosis and receive lower quality care for the same racist reasons.
    bwilson4web likes this.