Enhancing Efficiency of Retroviral Transduction of Host Cells

摘要

The present invention provides methods for enhancing transduction efficiency of a viral vector into a host cell such as an unstimulated stem cell. The methods involve transducing the host cell with the vector in the presence of an SAMHD1 inhibitor (e.g., a Vpx protein), and an inhibitor of mTOR complexes (e.g., rapamycin or analog compound thereof). Also provided in the invention are kits or pharmaceutical combinations for delivering a therapeutic agent into a target cell with enhanced targeting frequency and payload delivery. The kits or pharmaceutical combinations typically contain a viral vector encoding the therapeutic agent, an SAMHD1 inhibitor or a polynucleotide encoding the SAMHD1 inhibitor, and an inhibitor of mTOR complexes.

主权项

1. A method for enhancing transduction efficiency of a viral vector into a host cell, comprising transducing the host cell with the viral vector in the presence of (1) an mTOR inhibitor compound and (2) an inhibitor of SAM domain and HD domain-containing protein 1 (SAMHD1).