Pre-Selective Anti-HIV Vectors for Improved HIV Gene Therapy

Abstract

Lentiviral vector and method to pre-select/sort anti-HIV gene transduced cells prior to clinical transplantation.

Full Description

Current HIV gene protocols including stem cell gene therapy have not demonstrated any efficacy in clinical trials due to low transduction efficiencies and low in vivo gene marking. Currently used marking methods include the use of EGFP, which is used as a reporter gene to track transduced cells or to sort cells by flow cytometry. EGFP is not a natural protein and can be recognized as a foreign antigen and transduced cells get rejected.

Researchers at the University of California, Davis have developed a novel anti-HIV lentivirus vector that expresses a selective cell surface marker to purify the transduced cells. Expression of this selective marker on the surface of the transduced hematopoietic stem cells gives these cells a unique cell surface signature which could be used to distinguish them from non-transduced cells. This selective marker is not found on the surface of CD34+ HSCs. Therefore, for HIV stem cell gene therapy these vectors can be used to pre-select or sort the anti-HIV gene transduced cells prior to clinical transplantation. The transduced cells can be purified by immunomagnetic bead separation to obtain a pure or enriched population of anti-HIV transfected cells.

Applications

• Use of pure or enriched population of hematopoetic stem cells for transplantation into HIV- infected patients

Features/Benefits

• Selective marker is a natural human protein and will not be recognized as a foreign antigen and transduced cells will not get rejected
• Allow for the isolation of and delivery of a pure or enriched population of anti-HIV gene transduced cells into patients

Patent Status