Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy

US6268213A · kind A · utility

Inventors

• Richard Jude Samulski · Chapel Hill, US
• Christopher E. Walsh · Bethesda, US
• Arthur W. Nienhuis · Memphis, US
• Johnson M. Liu · Rockville, US
• Jeffrey L. Miller · Vancouver, US

Key dates

• Filing date: Jun 7, 1995
• Grant date: Jul 31, 2001
• Expiry date: Jun 7, 2015

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2710/10343
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The subject invention concerns a recombinant adeno-associated virus vector characterized as being capable of delivering and expressing at least one mammalian gene into a genome of a mammalian host cell such that the expression of the gene is regulated in a tissue specific manner by cis-acting regulatory and promoter elements of the gene. A method of using this recombinant adeno-associated virus vector for therapeutic purposes is also provided.