Prime editing to correct disease causative mutations in patient-derived Gaucher iPSC lines
Prime editing to correct disease causative mutations in patient derived Gaucher iPSC lines Lysosomes are the recycling bins of our cells, but what happens if they stop working? Do we get sick? Why would they stop working? Dysfunctions in lysosomes can be due to genetic mutations, changes in our genome that can lead to brain disease and premature death. Recently, the CRISPR/Cas9 system, that works