AAV-based conditional expression system

US11926841B2 · kind B2 · utility

Inventors

• Ulrich Koszinowski · Dießen am Ammersee, DE
• Simona Langer · Munich, DE
• Zsolt Ruzsics · Dießen am Ammersee, DE
• Christian Thirion · Nogent-sur-Oise, FR

Key dates

• Filing date: Sep 22, 2021
• Grant date: Mar 12, 2024
• Expiry date: Oct 22, 2041

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2830/002
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The present invention relates to a cell comprising (aa) a nucleic acid comprising in 5′ to 3′ direction (i) at least one adeno-associated virus (AAV) inverted terminal repeat (ITR) sequence; (ii) a promoter which is capable of being activated by (a) helper polypeptide(s) and optionally (a) helper polynucleotide(s); and (iii) a transgenic coding sequence under the control of said promoter of (aa)(ii); and (ab) a nucleic acid comprising in 5′ to 3′ direction (i) a promoter which is capable of being activated by said helper polypeptide(s) and optionally said helper polynucleotide(s); and (ii) at least one AAV rep gene coding sequence under the control of said promoter of (ab)(i); wherein said cell does not comprise an AAV cap gene and/or is not able to express any AAV cap gene product.