The day-in-life of your processor.

Picture by #Apple; from #IRDS #Roadmap 2022 (https://irds.ieee.org/editions/2022/executive-summary)

@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

#RPGR mutations cause inherited retinal dystrophies (#IRDs); the leading cause of #VisualLoss in children & working adults. We show RPGR regulates the disassembly of these #actin filaments where discs form and patient mutations prevent this, leading to reduced actin disassembly. /4