Liver biopsy confirmed base editing corrected a disease-causing SERPINA1 mutation in a living human liver, offering stronger evidence than blood tests alone.
Oh this is huge stuff that will pay off for a lot of conditions in coming years, it’s just unfortunately not a sign that there is going to be an available treatment for this condition in the pipeline.
Oh this is huge stuff that will pay off for a lot of conditions in coming years, it’s just unfortunately not a sign that there is going to be an available treatment for this condition in the pipeline.