Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell

US8361982B2 · kind B2 · utility

Assignees

• CALIFORNIA INSTITUTE OF TECHNOLOGY · US
• The Regents of the University of California · US

Inventors

• Carlos Lois-Caballe · Cambridge, US
• David Baltimore · Pasadena, US
• Xiao-Feng Qin · Saginaw, US
• Irvin S. Y. Chen · Rancho Palos Verdes, US
• Dong Sung An · Los Angeles, US

Key dates

• Filing date: Apr 28, 2010
• Grant date: Jan 29, 2013
• Expiry date: Mar 17, 2031

Classification

• Technology area (CPC Y): Emerging Cross-Sectional Technologies
• CPC primary: Y02A50/30
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG. 1A). Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of downregulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of downregulating CCR5, that are relatively non-cytotoxic to cells.