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#genetictherapy — Public Fediverse posts

Live and recent posts from across the Fediverse tagged #genetictherapy, aggregated by home.social.

  1. @gaetan_burgio I suspect this is going to be the 'long game' challenge for somatic in vivo #GenomeEditing field in tissues where they isn't a selective advantage to correction (i.e. metabolic)- how much is enough to meet your clinical endpoints?

    But the headway made for each of these trials in terms of demonstrating safety are still hugely important...In cases like #LCA #IRDs you may also need a drug-based intervention to slow death in unedited cells.

    #CRISPR #GeneticTherapy #GeneticDisease