High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia.

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ID: 77274
2019
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Abstract
Cystic fibrosis (CF) is a monogenic disorder caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Mortality in CF patients is mostly due to respiratory sequelae. Challenges with gene delivery have limited attempts to treat CF using in vivo gene therapy, and low correction levels have hindered ex vivo gene therapy efforts. We have used Cas9 and adeno-associated virus 6 to correct the ΔF508 mutation in readily accessible upper-airway basal stem cells (UABCs) obtained from CF patients. On average, we achieved 30%-50% allelic correction in UABCs and bronchial epithelial cells (HBECs) from 10 CF patients and observed 20%-50% CFTR function relative to non-CF controls in differentiated epithelia. Furthermore, we successfully embedded the corrected UABCs on an FDA-approved porcine small intestinal submucosal membrane (pSIS), and they retained differentiation capacity. This study supports further development of genetically corrected autologous airway stem cell transplant as a treatment for CF.
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vaidyanathan2019highefficiencycell Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Vaidyanathan, Sriram;Salahudeen, Ameen A;Sellers, Zachary M;Bravo, Dawn T;Choi, Shannon S;Batish, Arpit;Le, Wei;Baik, Ron;de la O, Sean;Kaushik, Milan P;Galper, Noah;Lee, Ciaran M;Teran, Christopher A;Yoo, Jessica H;Bao, Gang;Chang, Eugene H;Patel, Zara M;Hwang, Peter H;Wine, Jeffrey J;Milla, Carlos E;Desai, Tushar J;Nayak, Jayakar V;Kuo, Calvin J;Porteus, Matthew H;
Journal cell stem cell
Year 2019
DOI
S1934-5909(19)30457-6
URL
Keywords
cell therapy genome editing cystic fibrosis crispr cftr f508del cas9 cf airway stem cells basal cells

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