The California Human Induced Pluripotent Stem Cell Derivation & Characterization Center

Funding Type: 
hiPSC Derivation
Grant Number: 
ID1-06560
Investigator: 
ICOC Funds Committed: 
$0
Public Abstract: 
It has recently been appreciated that skin or blood cells from a patient can be “reprogrammed” back to a stage of development that emulates an embryonic stem cell, i.e., a cell that can yield the broad range of cell types in the human body. The great advantage of this technology is that the cells that come from these “induced” stem cells carry the genes of the diseased patient &, hence, may allow scientists to study that disease “in-a-dish”. Such stem cells are particularly valuable for obtaining cell types responsible for a given disease process that are otherwise inaccessible from a living patient, e.g., brain or heart cells. Once these cells are grown in a dish, they can be examined for targets that might arrest/reverse the disease process. The cells can even be used to discover new drugs against that target. Sanford-Burnham’s Stem cell Core (SCC) has great success in making these human induced pluripotent stem cells (hiPSCs) from patients’ skin cells. In fact, it presently produces hiPSCs for most of San Diego. We propose to become the hiPSC generating facility for California & derive hiPSCs from 3000 patients with a range of diseases using a technique that is safe & effective. To increase the throughput & decrease the cost of this process, the SCC will employ a computer-controlled laser system. It can also leverage its home institute’s extensive core facilities, its commercial collaborations, & the complementary expertise of its partner institutions in La Jolla.
Statement of Benefit to California: 
It has recently been appreciated that skin or blood cells from a patient can be “reprogrammed” back to a stage of development that emulates an embryonic stem cell, i.e., a cell that can yield the broad range of cell types in the human body. The great advantage of this technology is that the cells that come from these “induced” stem cells carry the genes of the diseased patient &, hence, may allow scientists to study that disease “in-a-dish”. Such stem cells are particularly valuable for obtaining cell types responsible for a given disease process that are otherwise inaccessible from a living patient, e.g., brain or heart cells. Once these cells are grown in a dish, they can be examined for targets that might arrest/reverse the disease process. The cells can even be used to discover new drugs against that target. Such drugs could dramatically reduce the financial & human cost of heretofore untreatable/incurable diseases; Californians would be the 1st beneficiaries of such drugs. In addition, the revenue from such drugs, based on licensing fees, royalties, & taxes, would ultimately return to California coffers, compensating the State for its investment in Prop. 71. Disease-in-a-dish technology will bring biotech companies to the State. Sanford-Burnham’s Stem Cell Core, which has great success in making these human induced pluripotent stem cells (hiPSCs) from patients’ skin cells, proposes to generate hiPSCs from 3000 patients with a range of diseases that plague Californians.

© 2013 California Institute for Regenerative Medicine