Description:
iPSC Reprogramming Challenges
Induced pluripotent stem cells (iPSCs) are a type of pluripotent stem cell generated directly from adult cells that can propagate indefinitely to give rise to every other cell type in the body. iPSCs are a valuable tool to study human development and disease. They are also useful for drug discovery and the development of cellular transplantation therapies, especially for genetic diseases. Despite improvements in reprogramming methods, the reprogramming efficiency remains low, and the risk of mutations is high. The need for genetic correction as a separate step during GMP production increases costs.
High Quality & Integration-free iPSC
A University of Colorado research group led by Drs. Ganna Bilousova and Igor Kogut has developed an RNA-based approach to reprogram human primary cells into high quality, integration-free iPSCs. The approach uses in vitro transcribed messenger RNAs synthesized with modified nucleotides (modified mRNAs) encoding pluripotency factors (SOX2, KLF4, cMYC, LIN28A, NANOG, OCT4-MyoD fusion) and mature embryonic stem cell-specific microRNA-367/302 mimics. The resulting iPSC colonies can then be isolated and directly expanded in feeder-free conditions.
Second generation technology allows concurrent gene correction during cellular reprogramming, decreasing expected costs of production. This technology is currently in development for the treatment of Epidermolysis bullosa, a genetic skin blistering disease, and is applicable for correction of other genetic diseases.
Advantages:
•100% success rate for iPSC generation, including difficult to reprogram, aged and senescent fibroblasts;
•Cost effective, fast, and efficient
Download Summary Document Here:
https://cuamc.technologypublisher.com/files/sites/cu3555h_cu4593h_combined_ipsc_reprogramming_and_gene_correction_ncs_20231.pdf