University of Florida Research Foundation, Incorporated

University of Pennsylvania

Lakshmanan Govindasamy

Sergei Zolotukhin

Mavis Agbandje-McKenna

Arun Srivastava

Li Zhong

George Vladimirovich Aslanidi

Kim M Van Vliet

James M Wilson

Jenny Agnes Sidrane

Mavis Agbandje-Mckenna

Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B.

Gene therapy for treating Wilson's disease

Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.

Capsid-modified rAAV vector compositions and methods therefor

Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.

Capsid modified rAAV vectors and methods of use

Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more symptoms of disease or abnormal conditions via in situ and/or ex vivo mammalian gene therapy methods. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.

Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use

Capsid-modified rAAV vectors and methods of use

Transduction efficiency, capsid-modified rAAV vectors and methods of use

Therapeutic methods based on tyrosine-substituted, capsid-modified rAAV vectors

Tyrosine-modified recombinant rAAV vector compositions and methods for use

Growing community of inventors

Gainesville, FL, United States of America

Lakshmanan Govindasamy