Disease Focus: Immune Disease
Embryonic stem cell-derived thymic epithelial cells
The function of the immune system throughout life is essential for protection from infections and cancer. T lymphocytes are white blood cells that choreograph the multiple responses that the body uses to control infection. T lymphocytes are produced in the thymus, a specialized organ located in the chest in front of the heart. The production […]
Genetic modification of the human genome to resist HIV-1 infection and/or disease progression
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 […]
HPSC based therapy for HIV disease using RNAi to CCR5.
RNA interference is a naturally occurring means to block the function of genes in our body. We propose that RNA interference can be used to block HIV-1 infection and its reproduction within the body. When RNA interference is introduced into a stem cell, its blocking activity will be present throughout the lifetime of the stem […]
ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry to a cell. Building on this observation, a study published in 2009 reported a potential “cure” in an AIDS patient with leukemia after receiving a […]
Stem Cell Cure for “Bubble Baby” Disease (SCID), Pioneered by UCLA’s Don Kohn
On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure for “bubble baby” disease, or severe combined immunodefiency (SCID). Babies born with SCID lack an immune system and have no ability to fight off infections so even a minor cold could be deadly. […]
Blood cells from Embryonic Stem Cells: Cornelis Murre – CIRM Science Writer’s Seminar
Cornelis Murre, Ph.D., spoke at the Scientific Writer’s Seminar, a workshop presented on September 17, 2008 at CIRM headquarters in San Francisco. Murre has a CIRM grant to develop an approach that would allow large-scale production of blood cells from human embryonic cells. Murre is a professor of biology at the University of California, San […]