The University of Kansas Medical Center Research Institute, Inc. (KUMCRI) was awarded a $642,400 Project Grant from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837). The grant, effective August 10, 2024 through April 30, 2028, supports the development of novel gene editing approaches for treating cystic fibrosis (CF).
Specifically, the project aims to use the CRISPR-based Homology-Independent Targeted Integration (HITI) method to permanently correct the genetic defect in the CFTR gene responsible for CF. KUMCRI will leverage an improved adeno-associated virus (AAV) vector, AAV2.5T, to efficiently deliver the HITI gene editing components to airway epithelial cells, including basal stem cells, in both in vitro cultures and a CF ferret model. The goal is to restore functional CFTR expression and correct the CF phenotype, providing a potential curative therapy for the disease.
As a sub-award under this project, the University of Iowa will develop the HITI gene editing approach and evaluate its efficacy in reconstituting CFTR in CF airway cells and the CF ferret lung.
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