Stem cell-therapy of recessive dystrophic epidermolysis bullosa (rdeb)
Faezeh Nasrollahi nia
,1,* Sanaz alioghli
,2 Somayyeh abbaszadeh
,3 Atena tazedel
,4 Elham ghorbani
,5 Milad feizollahi
1. department of biology, factuly of science, university mohaghegh ardabili
2. department of biology, factuly of science, university mohaghegh ardabili
3. department of biology, factuly of science, university mohaghegh ardabili
4. department of biology, factuly of science, university mohaghegh ardabili
5. department of biology, factuly of science, university mohaghegh ardabili
6. department of biology, factuly of science, university mohaghegh ardabili
Stem cells have high growth potential and are first defined by two major attributes: self-renewal and the ability to differentiate into one or more categories. mesenchymal stem cells, which have a potential for differentiation of chondrocytes, osteoblasts, adipocytes, fibroblasts, bone marrow stroma, and other tissues of mesenchymal origin, are capable of producing specific types of cells for these tissues in numerous tissues in the extrinsic living creature. mesenchymal stem cells have been considered by many researchers because of their unique characteristics for cell therapy programs. epidermolysis bullosa represents a family of severe, life threatening skin disorders resulting from mutations in genes encoding protein components of the cutaneous basement membrane zone. epidermolysis bullosa have been classified into the following types: epidermolysis bullosa simplex, junctional epidermolysis bullosa and dystrophic epidermolysis bullosa. although some forms, such as the junctional type, are lethal in the neonatal period, others, such as the dystrophic forms, lead to years of painful skin blistering and mutilating scarring. the most severe form of dystrophic epidermolysis bullosa is caused by recessive mutations in the type vii collagen gene (col7a1). the incidence of this disease is 1 in 50,000. percentage of probability is equal to all men and women of different races of mankind. skin cancer is more likely to develop than other people. symptoms of this disease are common, snoring, coughing, or other breathing problem, hair loss, blistering near the eyes and nose, blistering your mouth and throat and causing difficulty in eating and swallowing, bleaching the skin after minor damage or temperature change, a blisters at birth, dental problems such as rot, the presence of mildew (small white wings), loss or deformation of the nail.the aim of this study is investigate application of stem cell-therapy on recessive dystrophic epidermolysis bullosa (rdeb).
To this purpose, we conducted extensive library research and compiled the latest reports about application of stem cell-therapy on recessive dystrophic epidermolysis bullosa (rdeb).
currently, there is no deﬁnitive treatment for recessive dystrophic epidermolysis bullosa (rdeb), although recent progress in understanding the molecular basis of rdeb has provided a foundation for the development of cell- and gene-based therapies. however, gene therapy remains a challenge both because of the inability to identify and target stem cells that can assist in reconstituting all of the components of the skin, and the low efﬁciency of introducing the gene of interest into those cell. induced pluripotent stem cells (ipscs) are well-suited for gene and cell therapy because of their origin, self-renewal capability, and pluripotency, as well as their potential for gene correction. to develop ipsc-based therapy for skin diseases, the ability to generate keratinocytes from ipscs is a crucial prerequisite. the reprogramming of a patient’s somatic cells into ipscs is a promising new approach to establish human models for studying disease mechanisms, testing drugs, and developing cell therapies. in recent articles reported the spontaneous differentiation of ipscs into keratinocytes, as well as the directed differentiation of mouse ipscs into keratinocytes, evidence for directed differentiation of human ipscs into keratinocytes has not yet been reported. the main ﬁndings of the studies are that wild-type marrow-derived cells can migrate to the skin lesions seen in rdeb, produce col7 protein and anchoring ﬁbrils, prevent blister formation, and extend survival in a murine model of rdeb. hematopoietic cell transplantation is a treatment of choice due to the ability of engrafting cells to provide a life-long source of the deﬁcient enzyme that can be taken up by recipient’s cells. on closer inspection, however, col7 is not a “true” structural protein as it does not contribute to the cellular structure.
Although hematopoietic cell transplantation is not without its own complications, treatment with wild-type bone marrow cells is potentially more advantageous than other possible forms of rdeb therapy in several respects.it is shown that infusion of wild-type bm cells results in expression of col7, formation of anchoring ﬁbrils, and healing of the blister.
Stem cell, ipscs, rdeb,