Adenovirus vectors 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.
Claims25
An adenovirus-based gene therapy vector comprising the genome of an adenovirus 2 serotype in which the E1a and E1b regions of the genome, which are involved in early stages of viral replication, have been deleted and replaced by genetic material of interest.
An adenovirus-based gene therapy vector comprising adenovirus inverted terminal repeat nucleotide sequences and the minimal nucleotide sequences necessary for efficient replication and packaging and genetic material of interest.
An adenovirus-based gene therapy vector comprising an adenovirus genome which has been deleted for all E4 open reading frames, except open reading frame 6, and additionally comprising genetic material of interest.
An adenovirus-based gene therapy vector comprising an adenovirus genome which has been deleted for all E4 open reading frames, except open reading frame 3, and additionally comprising genetic material of interest.
A method for treating or preventing cystic fibrosis in a patient comprising administering to the pulmonary airways of the patient, a gene therapy vector comprising DNA encoding cystic fibrosis transmembrane conductance regulator.
Dependencies are inferred from the claim text.