Recent Patents on MicroRNA-Induced Pluripotent Stem Cell Generation

摘要

Regenerative medicine using pluripotent stem cells represents the future for curing developmental abnormalities, degenerative disorders and aging-related illnesses. However, today's regenerative medicine faces two major challenges with respect to stem cells: their shortage in supply and uncertain safety in clinical therapy. The recent discovery of induced pluripotent stem cells (iPS cells or iPSCs) derived from patients' somatic cells presents a possible solution for the supply shortage problem. Yet, iPSC generation with the enforced overexpression of previously defined four factors Oct4-Sox2-Klf4-c-Myc (OSKM) also causes oncogenic stimulation, which leads to potential tumorigenicity. The more recent development of miR302-mediated iPSC generation may further solve this problem since miR-302, a tiny 23-nucleotide non-coding microRNA (miRNA), is able to replace all four OSKM in mediating iPSC formation while preventing the onset of stem cell tumorigenicity. MiR-302-reprpgrammed iPSCs (mirPSCs) not only possess a highly demethylated genome but also share >92% gene expression similarity with human embryonic stem cells (hESCs).