Phase I Trial of Intranasal and Endobronchial Administration of a Recombinant Adeno-Associated Virus Serotype 2 (rAAV2)-CFTR Vector in Adult Cystic Fibrosis Patients: A Two-Part Clinical Study

Abstract excerpt

Recombinant adeno-associated serotype 2-based vectors (rAAV2) possess a number of theoretical advantages for cystic fibrosis (CF) gene therapy because they elicit little or no inflammatory response and generally result in stable expression. rAAV2 vectors expressing the cystic fibrosis transmembrane conductance regulator (CFTR) gene have previously been shown to mediate stable correction of the CF defect in CF...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cystic Fibrosis Research Advances
• Share to Energy Harvesting in Wireless Networks
• Share to Virus-based gene therapy research