Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy

US6342390B1 · kind B1 · utility

Assignee

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

Inventors

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

Key dates

• Filing date: Nov 23, 1994
• Grant date: Jan 29, 2002
• Expiry date: Nov 23, 2014

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…