Compositions for treating cystic fibrosis
AssigneesUniversity of Florida Research Foundation, Inc., The Johns Hopkins University
InventorsTerence R. Flotte, Jeffrey R. Sirninger, William B. Guggino, Liudmila Cebotaru, Christian Müller
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Abstract
The invention relates to compositions and methods of treating cystic fibrosis. More specifically, this invention relates to the use the AAV vectors and constructs to provide gene therapy to cystic fibrosis patients.
Claims4
A recombinant adenoviral associated virus (AAV) gene therapy particle comprising a nucleic acid encoding a truncated cystic fibrosis transmembrane conductance regulator (CFTR) protein consisting of the amino acid sequence of SEQ ID NO 9, operatively linked to a promoter, and a first and a second AAV inverted terminal repeat (ITR) sequence flanking the sequence encoding the truncated CFTR protein.
Dependencies are inferred from the claim text.