Clinically Relevant iPS Cell Lines

Funding Type: 
New Cell Lines
Grant Number: 
RL1-00645
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
Studies outlined in this proposal are designed to provide a platform for research and development of new clinically translatable iPS cell lines specifically addressing the shortcomings of current iPS procedures that impede potential translation of this approach for use in the clinic. Induction of pluripotent status in somatic cells by directed reprogramming in-vitro, induced pluripotent stem (iPS) cells, is of great potential significance for the generation of disease and patient specific cell lines and cell therapy (1,2,3). In addition to the need for genetic transduction of factors using retroviral vectors, this procedures requires selection of iPS host cells using genetic transduction of selectable markers followed by drug selection to isolate iPS cells. Although quite promising and revolutionary, to date iPS techniques share several main shortcomings which collectively impede potential translation of this approach for use in the clinic. Our long-term goal is to develop, tools, and cell lines that allow translation of iPS cells into therapies for the clinic. We propose to eliminate the need for genetic alterations of iPS cells, increase the efficiency of the process, and develop clinically applicable identification methodologies for iPS cells. Human iPS cells hold great potential for the generation of disease and patient specific cell lines and cell therapy. The current requirements for genetic alterations, however, are among major barriers for this potential. Accomplishing the specific aims outlined in this proposal will provide the foundation required to assess the possibility of generation of Human iPS cell lines by clinically relevant methodologies, eliminating the current impediments for translation of iPS cells into the clinic.
Statement of Benefit to California: 
Studies outlined in this proposal are designed to provide a platform for research and development of new clinically translatable induced pluripotent stem (iPS) cell lines specifically addressing the shortcomings of current iPS procedures that impede potential translation of this approach for use in the clinic. We propose to eliminate the need for genetic alterations of iPS cells, increase the efficiency of the process, and develop clinically applicable identification methodologies for iPS cells. Human iPS cells hold great potential for the generation of disease and patient specific cell lines, cell therapies and tissue regeneration, benefiting California’s pressing unmet need for patient customized cell lines that could be used for treatment of life threatening diseases and injuries. Accomplishing the specific aims outlined in this proposal will provide the foundation required to assess the possibility of generation of Human iPS cell lines by clinically relevant methodologies, eliminating the current impediments for translation of iPS cell lines into the clinic.

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