In vitro correction of a mutation that causes autosomal dominant polycystic kidney disease with prime editing

Identifier:  TFG:6958

Handle: https://hdl.handle.net/20.500.11797/TFG6958

Authors:  Morcillo Castro, Alba

Abstract:
Autosomal dominant polycystic kidney disease is an inherited disease caused primarily by mutations in the PKD1 gene. The hypothesis is that prime editing is capable of correcting a mutation causing ADPKD in cells derived from a mouse model of the disease. To test this hypothesis, lipofectamine was optimized as a plasmid transfection reagent and the prime editing machinery was introduced with this reagent. It was not possible to correct the mutation with the guides that were tested, so more studies are still necessary in which other guides and other experimental designs could be tested.