Efficient AAV vectors
AssigneeThe Regents of the University of California
InventorsJian-Yun Dong, Yuet Wai Kan
Life of this patent
Estimating the term…
Abstract
The present invention provides improved AAV vectors for delivery of therapeutic nucleic acids. In particular, it provides vectors for delivery of CFTR polynucleotides.
Claims12
An AAV vector comprising a transcription cassette containing a nucleic acid of interest operably linked to a promoter, an AAV ITR sequence, and which has less than about 10 non-ITR AAV nucleotides.
An expression cassette comprising a truncated CFTR polynucleotide encoding a functional CFTR polypeptide, wherein said polynucleotide is operably linked to a promoter and has a deletion in the cytoplasmic tail domain or the regulatory domain.
An AAV vector which has less than about 10 non-ITR AAV nucleotides, wherein said vector is synthesized using overlapping PCR primers that hybridize to the entire right-hand ITR region or the entire left-hand ITR region.
Dependencies are inferred from the claim text.