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

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Grant Award Details

Grant Type:
SEED Grant
Grant Number:
RS1-00172
Investigator(s):
Name:
Dr. Irvin SY Chen
Institution:
University of California, Los Angeles
Type:
PI

Disease Focus:
HIV/AIDS, Immune Disease, Infectious Disease
Human Stem Cell Use:
Embryonic Stem Cell
Award Value:
$616,800
Status:
Closed

Progress Reports

Reporting Period:
Year 2

Grant Application Details

Application Title:

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

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.

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