@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