Summary
Researchers in the UCLA Departments of Medicine and Microbiology, Immunology and Molecular Genetics have developed retroviral vectors pseudotyped with a modified Sinbus virus envelope that exhibit reduced tropism and can be used for the targeted transduction of heterologous genes into cells.
Background
There have been numerous attempts to develop targeted gene transduction systems based upon viral vectors, including lentiviral vectors. Many of these attempts have had limited success due to inconsistent tissue specificity. Lentiviral vectors with modified envelope proteins have shown increased specific binding activity, but these vectors have low fusion activity, leading to inefficient cell entry. Although the Sindbis virus contains an envelope protein that maintains viral infectivity after being substantially modified, the Sindbis viral vector itself is cytotoxic. This toxicity can be avoided by pseudotyping lentiviral vectors with modified Sindbus virus envelopes. These pseudotyped vectors, however, continue to exhibit residual non-specific tropism.
Innovation
Researchers in the UCLA Departments of Medicine and Microbiology, Immunology and Molecular Genetics have developed retroviral vectors pseudotyped with a modified Sinbus virus envelope that exhibit reduced tropism and can be used for the targeted transduction of heterologous genes into cells. The Sinbis virus envelopes can pseudotype specific lentiviral and murine retroviral vectors. These vectors maintain high viral titer and have applicability to a wide range of gene therapies.
Applications
▶ Targeted heterologous gene transduction
▶ Other forms of gene therapy
▶ Diagnosis and research
Advantages
▶ Stable
▶ High viral titers
▶ Targeted cell specific transduction
▶ Limited immunogenicity
State Of Development
▶ Patent 9,163,248 issued on October 20, 2015 and is assigned to the Regents of the University of California