Methods and composition for in vivo gene therapy

US5827703A · kind A · utility

Assignee

• The Regents of the University of California · US

Inventors

• Robert James Debs · Mill Valley, US
• Ning Zhu · 安丰镇, CN

Key dates

• Filing date: May 19, 1994
• Grant date: Oct 27, 1998
• Expiry date: May 19, 2014

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K39/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Novel methods and compositions are provided for introducing a gene capable of modulating the genotype and phenotype into two or more tissues following systemic administration. The gene can be introduced into a mammalian host by way of an expression vector either as naked DNA or complexed to lipid carriers, particularly cationic lipid carriers. Multiple individual tissues can be transfected using naked DNA. Using a DNA: lipid carrier complex, multiple tissues and cell types can be transfected. The techniques and compositions find use in the palliation or treatment of any of a variety of genetic-based disorders.