Viral Expression Vector Targeting HIV-1

Summary

UCLA researchers from the Department of Microbiology, Immunology, & Molecular Genetics have developed a viral expression vector that combines two reagents effective against HIV-1 infection.

Background

Highly active antiretroviral therapy (HAART) has decreased the morbidity and mortality of human immune deficiency virus (HIV) in all parts of the world.  The effectiveness of this life-long treatment however is limited by the side effects and long term toxicities of the medications, their cost, and the often rapid development of drug resistance.  Hematopoietic progenitor/stem cell (HPSC) based approaches may provide a continuous means of controlling HIV after a single or infrequent doses.

Innovation

UCLA researchers have developed a new viral expression vector capable of inhibiting binding of HIV to the cell and preventing HIV fusion into the cell.  By targeting two different aspects of HIV infection, this combination viral expression vector increases treatment efficacy.  The vector targets both CCR5, the primary HIV-1 co-receptor, as well as C46, an HIV entry inhibitor.  Combination therapy protects from diverse strains of HIV and to prevent the emergence of drug resistant mutations.  This new approach holds great promise as a one time or infrequent therapy that can eliminate the need for HAART.  C46 and lentiviral vector transduction have been shown to be safe and non-immunogenic through human and antiviral studies.

Applications

• HIV gene therapy

Advantages

• Single of infrequent dose needed
• Dual targets of action
• Potentially curative
• Eliminates need for lifelong HAART drugs
• Reduced cost of lifetime treatment
• Can reconstitute AIDS damaged immune systems

Patent Status

Additional Patents Pending