Hematopoetic Stem Cell Transplantation and Genotyping in Cystic Fibrosis Mouse Model

Introduction: Although CF has been a target of gene therapy for decades, no viable approach is proven successful so far. Here, we attempted transplantation of hematopoietic stem and precursor cells (HSPCs) containing wild-type Cftr into Cftr-deficient mice to study restoration of CFTR function especially in non-epithelial cells. To accomplish this, we transplanted bone marrow from the wild-type BL6 and Cftr deficient mice into Cftr deficient mice to generate chimeras, namely CFB6 and CFCF. In fact, CFB6 mice showed significant increased survival upon Pseudomonas aeruginosa airway infection compared to CFCF mice.…