Liver biopsy confirmed base editing corrected a disease-causing SERPINA1 mutation in a living human liver, offering stronger evidence than blood tests alone.
Look I’m just out here hoping it works for other organs eventually. It is hard to express what this would mean for those of us suffering genetic maladies.
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.
Don’t get your hopes up if you or a loved one suffer from AATD but this is extremely cool.
Wow.
Look I’m just out here hoping it works for other organs eventually. It is hard to express what this would mean for those of us suffering genetic maladies.
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.