Chiron Corporation

Other

University of California

Novartis Vaccines & Diagnostics, Gmbh

Varavani J Dwarki

Ronald N Zuckermann

Michael A Innis

Fred Ehrenkranz Cohen

Tetsuo Uno

John E Murphy

Nathalie Dubois-Stringfellow

Jaime A Escobedo

Shang-Zhen Zhou

Martha B Ladner

Mervin C Yoder

Arun Srivastava

Katherine Rendahl

William C Manning, Jr

Xu-Shan Wang

Selvarangan Ponnazhagan

Robert H Chloemer

Fei Wang

Shangzhen Zhou

Sheldon S Miller

This invention relates compositions and methods for increasing the uptake of polynucleotides into cells. Specifically, the invention relates to vectors, targeting ligands, and polycationic agents. The polycationic agents are capable of (1) increasing the frequency of uptake of polynucleotides into a cell, (2) condensing polynucleotides; and (3) inhibiting serum and/or nuclease degradation of polynucleotides.

Compositions and methods for polynucleotide delivery

Gene delivery vectors, such as, for example, recombinant adeno-associated viral vectors, and methods of using such vectors are provided for use in treating or preventing diseases of the eye.

Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

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.

AAV vectors

Polycationic polymers

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.

Materials and methods for simplified AAV production

Growing community of inventors

Pittstown, NJ, United States of America

Varavani J Dwarki