Adenovirus vector for gene therapy
AssigneeGenzyme Corporation
InventorsRichard J. Gregory, Donna Armentano, Larry A. Couture, Alan E. Smith
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Abstract
Gene Therapy vectors, which are especially useful for cystic fibrosis, and methods for using the vectors are disclosed.
Claims19
An adenoviral vector comprising an adenovirus genome from which one or more of the E4 open reading frames has been deleted, but retaining sufficient E4 sequences to promote virus replication in vitro, and additionally comprising a DNA sequence of interest operably linked to expression control sequences and inserted into said adenoviral genome.
A method for providing cystic fibrosis transmembrane conductance regulator protein to airway epithelial cells of a cystic fibrosis patient comprising administering directly to airway epithelial cells of the patient an adenoviral vector, said vector comprising an adenovirus genome from which one or more E4 open reading frames has been deleted, but retaining sufficient E4 sequences to promote virus replication in vitro, and additionally comprising a DNA sequence encoding cystic fibrosis transmembrane regulator protein operably linked to expression control sequences and inserted into the E1 region said adenoviral genome, under conditions whereby the DNA sequence encoding cystic fibrosis transmembrane regulator protein is expressed and a functional chloride ion channel is produced in the airway epithelial cells of the patient.
Dependencies are inferred from the claim text.