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Thread: Gene editing fixes cystic fibrosis in mice

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    Gene editing fixes cystic fibrosis in mice

    Gosh I hope human trials are underway and are successful. It's a terrible disease.

    See:
    https://www.nature.com/articles/d415...?sf212689637=1


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    Thank you for sharing the reasons that curiosity awakens discovery. Looking at what solutions might be discovered awakens a bit of charity and a wish of hope.

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    This piece adds to how solutions add to reason to stay connected to the each day's search.

    In one remarkable case in 2017, this approach was used to treat a young boy whose life was in grave danger after he lost nearly 80% of his skin to the disease. De Luca and his team harvested epidermal stem cells from the child, used a viral vector to insert a correct copy of the mutant gene, and grew skin grafts from the altered stem cells. Once this lab-grown skin was grafted on to the boy’s body, it behaved like healthy skin and showed no signs of blistering.


    De Luca stresses that unlike treatments for ADA-SCID and leukodystrophy, this is not a cure for the disease. “We are just taking care of some of the tissues that are involved in the disease by making a gene correction.”


    Correcting a genetic blip Sickle cell disease has become a prime target for gene therapy, because by simply swapping one amino acid for another in the haemoglobin gene, millions could avoid the debilitating chronic anaemia that results from the disorder.


    Preclinical research in mice has shown that CRISPR–Cas9 gene-editing technology can be used to correct the mutated haemoglobin gene in 20–25% of haematopoietic stem cells. Mark Walters of the blood and bone marrow transplant programme at the UCSF Benioff Children’s Hospital in the US says this is almost certainly enough to have strong clinical benefit.


    The next step is to test it in humans, and although the risks and side-effects are well known when transplanting a patient’s own cells back into their body, less is known about the potential ‘off-target’ effects of gene editing.


    “Besides this targeted sickle mutation, what other areas in the human genome, that’s so huge, might be targeted by the same reagents?” asks Walters.

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    sometimes without it




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