Blood Res.  2014 Mar;49(1):7-14. 10.5045/br.2014.49.1.7.

Disease modeling and cell based therapy with iPSC: future therapeutic option with fast and safe application

• Kim C

• KMID: 2172817
• DOI: http://doi.org/10.5045/br.2014.49.1.7

Abstract

Induced pluripotent stem cell (iPSC) technology has shown us great hope to treat various human diseases which have been known as untreatable and further endows personalized medicine for future therapy without ethical issues and immunological rejection which embryonic stem cell (hES) treatment has faced. It has been agreed that iPSCs knowledge can be harnessed from disease modeling which mimics human pathological development rather than trials utilizing conventional rodent and cell lines. Now, we can routinely generate iPSC from patient specific cell sources, such as skin fibroblast, hair follicle cells, patient blood samples and even urine containing small amount of epithelial cells. iPSC has both similarity and dissimilarity to hES. iPSC is similar enough to regenerate tissue and even full organism as ES does, however what we want for therapeutic advantage is limited to regenerated tissue and lineage specific differentiation. Depending on the lineage and type of cells, both tissue memory containing (DNA rearrangement/epigenetics) and non-containing iPSC can be generated. This makes iPSC even better choice to perform disease modeling as well as cell based therapy. Tissue memory containing iPSC from mature leukocytes would be beneficial for curing cancer and infectious disease. In this review, the benefit of iPSC for translational approaches will be presented.

Keyword

Stem cell; iPSC; Cell transplant; Patient specific medicine; Blood disorder

MeSH Terms

Cell Line
Communicable Diseases
Embryonic Stem Cells
Epithelial Cells
Ethics
Fibroblasts
Hair Follicle
Hope
Humans
Precision Medicine
Leukocytes
Memory
Pluripotent Stem Cells
Rodentia
Skin
Stem Cells
Transplants

Figure

• 

  Fig. 1 Patent specific iPSC disease modeling and cell based transplantation therapy. Mononuclear cells from patient are used for generating iPSC. Depending on the source of cells, both memory contained cells such as mature blood cell and memory lacking immature cell mediated iPSC are generated. Subsequent differentiation from patient specific iPSC can be directly used for personalized cell based therapy with proper cell lineages such as blood, muscle, and neuron. Gene editing technology such as ZFN, TALEN, and CRISPR will be utilized to fix the genetic error from affected patients even with large DNA deletions. Also, patient iPSC based personalized medicine to find right dosage of known medications or novel therapeutic agents can be tested.

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