Paper Title
Designing the Donor Construct for Knocking-In ABCB11-the Mutant Gene in Progressive Familial Intrahepatic Cholestasis

Abstract
PFIC2 (Progressive Familial Intrahepatic Cholestasis) is caused due to mutation in ABCB11 gene. Genome editing with engineered nucleases (GEEN) allows us to edit or correct genomes using artificially engineered nucleases, or "molecular scissors”.Here, we intend to develop genome editing tools, especially CRISPR-Cas9 based for correcting/improving the function of the bile saltexport pump which is defective in PFIC type-2.We planned a targeted insertion of ABCB11 gene in one of the “Safe-Harbor sites” in human genome, the AAVS1 site. We cloned ABCB11, the Gene of Interest (GoI) into a vector where the GoI is flanked on both sides by AAVS1 sequences present in the human genome to facilitate homologous recombination.