Abstract: Provided are methods for selectively expressing therapeutic molecules, such as secretory proteins, antisense molecules and ribozymes, in the liver. The methods find use in treating hepatic diseases or conditions. The methods also find use in treating any disease or condition in which systemic administration of the therapeutic substance, for example, a secretory protein, is desired. The methods involve administering to a mammalian patient having a need for liver expression of a therapeutic molecule an AAV vector containing a therapeutically effective amount of the therapeutic molecule. Also provided are novel vectors employable in these methods.

Abstract: The present invention is directed to a recombinant adenovirus vector comprising two inverted terminal repeats (ITRs) each of which comprises a D-sequence having (i) from 5 to 15 native nucleotides and (ii) one or more deletions or substitutions therein.

Abstract: The invention provides a method for producing purified replication-defective recombinant AAV virions. The method comprises introducing into a suitable host cell an AAV vector, an AAV helper construct and an adenoplasmid accessory construct into the host cell. The adenoplasmid accessory plasmid is composed adenovirus plasmid DNA unable to be packaged into adenoviral particles because it lacks packaging signal sequence(s) or it contains additional sequences making it too large to package. The host cell is cultured to produce crude rAAV virions and then lysed. The resulting cell lysate is applied to a chromatographic column containing sulfonated cellulose or subjected to cesium chloride equilibrium gradient centrifugation and the purified rAAV virions are recovered.

Abstract: Provided are methods for selectively expressing therapeutic molecules, such as secretory proteins, antisense molecules and ribozymes, in the liver. The methods find use in treating hepatic diseases or conditions. The methods also find use in treating any disease or condition in which systemic administration of the therapeutic substance, for example, a secretory protein, is desired. The methods involve administering to a mammalian patient having a need for liver expression of a therapeutic molecule an AAV vector containing a therapeutically effective amount of the therapeutic molecule. Also provided are novel vectors employable in these methods.

Abstract: The invention provides a method for producing purified replication-defective recombinant AAV virions. The method comprises introducing into a suitable host cell an AAV vector, an AAV helper construct and an adenoplasmid accessory construct into the host cell. The adenoplasmid accessory plasmid is composed adenovirus plasmid DNA unable to be packaged into adenoviral particles because it lacks packaging signal sequence(s) or it contains additional sequences making it too large to package. The host cell is cultured to produce crude rAAV virions and then lysed. The resulting cell lysate is applied to a chromatographic column containing sulfonated cellulose or subjected to cesium chloride equilibrium gradient centrifugation and the purified rAAV virions are recovered.

Abstract: The invention provides a method for producing purified replication-defective recombinant AAV virions. The method comprises introducing into a suitable host cell an AAV vector, an AAV helper construct and an adenoplasmid accessory construct into the host cell. The adenoplasmid accessory plasmid is composed adenovirus plasmid DNA unable to be packaged into adenoviral particles because it lacks packaging signal sequence(s) or it contains additional sequences making it too large to package. The host cell is cultured to produce crude rAAV virions and then lysed. The resulting cell lysate is applied to a chromatographic column containing sulfonated cellulose or subjected to cesium chloride equilibrium gradient centrifugation and the purified rAAV virions are recovered.