Adeno-associated virus materials and methods
OwnerPhilip R. Johnsoninventor
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Abstract
The present invention provides adeno-associated virus (AAV) materials and methods which are useful for DNA delivery to cells. More particularly, the invention provides recombinant AAV (rAAV) genomes, methods for packaging rAAV genomes, stable host cell lines producing rAAV and methods for delivering genes of interest to cells utilizing the rAAV. Particularly disclosed are rAAV useful in generating immunity to human immunodeficiency virus-1 and in therapeutic gene delivery for treatment of neurological disorders.
Claims25
A recombinant adeno-associated virus genome comprising adeno-associated virus inverted terminal repeats flanking DNA sequences encoding an immunodeficiency virus protein operably linked to promoter and polyadenylation sequences.
A recombinant adeno-associated virus genome comprising adeno-associated virus inverted terminal repeats flanking DNA sequences encoding a polypeptide selected from the group consisting of tyrosine hydroxylase, aromatic amino acid decarboxylase, nerve growth factor, brain derived neurotrophic factor, NT-3, NT-4/5, glial derived neurotrophic factor and fibroblast growth factor, wherein said DNA sequences are operably linked to promoter and polyadenylation sequences.
A mammalian host cell stably transfected with a recombinant adeno-associated virus genome and with adeno-associated virus rep-cap genes.
Dependencies are inferred from the claim text.