As mutations leading to herPAP can be distributed throughout the CSF2RA gene which results in PAP-phenotypes of varying severity21, we here evaluated the feasibility of a targeted integration of the codon-optimized version of the CSF2RA transgene (CSF2RAcoop) into the PPP1R12C gene intron 1, better known as AAVS1 locus, as a comprehensive therapeutic strategy. The gene discussed is CSF2RA; the disease is pulmonary alveolar proteinosis.