Methods of using adeno-associated virus rep protein

US6821511B2 · kind B2 · utility

Assignee

• The United States of America, as represented by the Department of Health and Human Services · US

Inventors

• Robert Kotin · Bethesda, US
• Brian Safer · Cumberland, US
• John A. Chiorini · Silver Spring, US
• Matthew Weitzman · Village of La Jolla, US
• Roland A. Owens · Arlington, US
• Stephen M. Wiener · Boston, US

Key dates

• Filing date: Aug 3, 2001
• Grant date: Nov 23, 2004
• Expiry date: Nov 1, 2021

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14162
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

A composition for delivering at least one DNA sequence encoding a desired protein or polypeptide (such as a therapeutic agent) to a cell. The composition comprises an adeno-associated virus rep protein (or a nucleic acid sequence encoding an adeno-associated virus rep protein) and a genetic construct including at least one DNA sequence encoding a protein or polypeptide or genetic transcript of interest and a promoter controlling the at least one DNA sequence. The genetic construct also includes a first adeno-associated virus ITR or protein or derivative thereof and a second adeno-associated virus ITR or a portion or derivative thereof. The first and second adeno-associated virus ITRs or portions or derivatives thereof flank the at least one DNA sequence encoding the protein or polypeptide or genetic transcript of interest and the promoter controlling the at least one DNA sequence encoding the protein or polypeptide or genetic transcript of interest. Such a composition provides for integration of genetic material at a specific locus in the human chromosome, while minimizing the possibility of inadvertent inactivation of host genes and minimizing the possibility of viral contaminatio…