Integration-free T cell-derived human induced pluripotent stem cells (iPSCs) from a patient with recessive dystrophic epidermolysis bullosa (RDEB) carrying two compound heterozygous mutations in the COL7A1 gene

Abstract

Expanded human T cells from a Japanese female with recessive dystrophic epidermolysis bullosa (RDBE) were used to generate integration-free induced pluripotent stem cells (iPSCs) by exogenous expression of four reprogramming factors, OCT3/4, SOX2, cMYC, KLF4, using Sendai virus vector (SeVdp). The authenticity of established iPSC line, RDEB-iPSC26, was confirmed by the expressions of stem cell markers and the differentiation capability into three germ layer. RDEB-iPSC26 may be a useful cell resource for the establishment of in vitro RDEB modeling and the study for developing gene and cell therapy.

Establishment of integration-free induced pluripotent stem cells from human recessive dystrophic epidermolysis bullosa keratinocytes

“…These groups used conventional retroviral vectors for iPSC induction. Conversely, several integration-free reprogramming systems have been described, such as SVV and episomal vectors, for the establishment of iPSCs [18,[26][27][28]. Very recently Shinkuma and colleagues succeeded in generating iPSCs from fibroblasts of a dominant dystrophic EB patient using episomal vectors and further corrected the responsible gene abnormality with the CRISPR/Cas9 system [29].…”

Inside out: regenerative medicine for recessive dystrophic epidermolysis bullosa

“…It also demonstrates that functional C7 is dispensable for stem cell renewal and differentiation into both hematopoietic and non-hematopoietic lineages. More recently, iPSCs have been derived from RDEB patient T cells (33). Although generating iPSCs from EB patients is an important first step, these cells still harbor the mutations in the COL7A1 gene.…”

Stem Cells in Cardiovascular Medicine: the Road to Regenerative Therapies

“…The important theme in these different reprogramming protocols is that they produce iPSCs without integrating vectors. Although these protocols are innovative and may be advantageous over existing protocols, the most efficient way to accomplish this goal currently is by using a nonintegrating Sendai virus as the vector for the reprogramming factors [52, 53]. Using Sendai virus for reprogramming is straightforward, has a high transduction efficiency, and is nonintegrating.…”