Genetic modification of the human genome to resist HIV-1 infection and/or disease progression

Funding Type: 
SEED Grant
Grant Number: 
Award Value: 
Disease Focus: 
Immune Disease
Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 

The proposed studies describe the genetic approaches utilizing human embryonic stem cells to suppress and/or eliminate the expression of the human protein CCR5. CCR5 is found on the surface of white blood cells. HIV-1 attaches to CCR5 and uses CCR5 to enter into its target cells. Our approach is to utilize established as well as new non-established approaches to prevent CCR5 from appearing on the surface of the cells. If CCR5 is not present, HIV-1 cannot infect the cells. Interestingly, this concept has already been proven in nature. Approximately 1% of the Caucasian population is genetically deficient for CCR5 and these individuals are resistant to HIV-1 transmission. Their white blood cells, when placed in culture, also resist HIV-1 infection in the laboratory. As such, we believe that our approach can be used to protect high risk individuals from HIV-1 infection as well as impede or stop progression of disease in those individuals already infected.

Statement of Benefit to California: 

According to the Centers for Disease Control, California is second only to New York of individuals living with AIDS. Developing means to stop HIV-1 infection and cure those individuals already infected with HIV-1 is of paramount importance for the state of California.

Progress Report: 

The overall goal of this project is to investigate the use of embryonic stem cells and blood stem cells as a potential therapeutic approach for HIV-1 disease. We investigated a process known as RNA interference to block HIV-1 infection. We characterize the properties of cells carrying RNA interference to HIV-1 and developed new tools to facilitate the study.