An optimized vector and cassette system expressing the inducing transcription factors to drive generation of iPS cells.

Stem cell generation can require multiple viral vectors to deliver the genes using multiple plasmids or lentiviral/retroviral vectors producing heterogeneous cultures and time-consuming QC and selection required for usable iPSCs.

A single adenoviral vector expressing the multiple inducing factors ensures in one "cassette" ensures the genes interact correctly to reprogram adult somatic cells into induced pluripotent stem (iPS) cells. The system eliminates the effects of insertional mutagenesis and greatly improves the efficiency of reprogramming. Rather than multiple vectors, it uses a single adenoviral vector to transport the transcription factors into the desired cell. This approach allows for homogeneous infectivity and a balanced expression of genes. Further, a system has been optimized to reprogram a patient's own cells to reprogram different types of stem cells into iPS cells.

Pre-Clinical Validation

• A single adenoviral vector for delivery of critical factors for inducing stem cells
• CMV promoters for each reprogramming factor can be optimized
• Production of iPS cells from adult somatic cells (any adult human cell)
• Method uses a non-insertional adenoviral vector (i.e. safer and less immunogenic)
• Rapid iPS cell reprogramming in three days.

Paul, Sharan et al. “Co-expression networks in generation of induced pluripotent stem cells.” Biology open vol. 5,3 300-10. 18 Feb. 2016, doi:10.1242/bio.016402

Publication Number: US-2014-0170752-A1
Patent Title: Generation of iPS Cells and Associated Methods
Jurisdiction/Country: United States
Application Type: Non-Provisional

Aaron Duffy
(801) 585-1377
aaron.duffy@utah.edu