Generation of induced pluripotent stem cells as a potential source of hematopoietic stem cells for transplant in PNH patients

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia caused by lack of CD55 and CD59 on blood cell membrane leading to increased sensitivity of blood cells to complement. Hematopoietic stem cell transplantation (HSCT) is the only curative therapy for PNH, however, lack of HLA-ma...

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Podrobná bibliografie
Vydáno v:	Annals of hematology Ročník 95; číslo 10; s. 1617 - 1625
Hlavní autoři:	Phondeechareon, Tanapol, Wattanapanitch, Methichit, U-pratya, Yaowalak, Damkham, Chanapa, Klincumhom, Nuttha, Lorthongpanich, Chanchao, Kheolamai, Pakpoom, Laowtammathron, Chuti, Issaragrisil, Surapol
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	Berlin/Heidelberg Springer Berlin Heidelberg 01.10.2016 Springer Nature B.V
Témata:	Animals Antigens, CD34 - biosynthesis CD55 Antigens - biosynthesis CD59 Antigens - biosynthesis Cellular Reprogramming Techniques Embryoid Bodies Female Fibroblasts - cytology Hematology Hematopoietic Stem Cell Transplantation Hemoglobinuria, Paroxysmal - therapy History, 16th Century History, 17th Century Humans Induced Pluripotent Stem Cells - cytology Induced Pluripotent Stem Cells - transplantation Leukosialin - biosynthesis Medicine Medicine & Public Health Mice, Inbred BALB C Mice, Nude Oncology Original Article Skin - cytology Teratoma - pathology Transplantation, Autologous Hematopoietic cell differentiation Paroxysmal nocturnal hemoglobinuria Induced pluripotent stem cells Hematopoietic stem cells
ISSN:	0939-5555, 1432-0584, 1432-0584
On-line přístup:	Získat plný text
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Popis
Shrnutí:	Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia caused by lack of CD55 and CD59 on blood cell membrane leading to increased sensitivity of blood cells to complement. Hematopoietic stem cell transplantation (HSCT) is the only curative therapy for PNH, however, lack of HLA-matched donors and post-transplant complications are major concerns. Induced pluripotent stem cells (iPSCs) derived from patients are an attractive source for generating autologous HSCs to avoid adverse effects resulting from allogeneic HSCT. The disease involves only HSCs and their progeny; therefore, other tissues are not affected by the mutation and may be used to produce disease-free autologous HSCs. This study aimed to derive PNH patient-specific iPSCs from human dermal fibroblasts (HDFs), characterize and differentiate to hematopoietic cells using a feeder-free protocol. Analysis of CD55 and CD59 expression was performed before and after reprogramming, and hematopoietic differentiation. Patients’ dermal fibroblasts expressed CD55 and CD59 at normal levels and the normal expression remained after reprogramming. The iPSCs derived from PNH patients had typical pluripotent properties and differentiation capacities with normal karyotype. After hematopoietic differentiation, the differentiated cells expressed early hematopoietic markers (CD34 and CD43) with normal CD59 expression. The iPSCs derived from HDFs of PNH patients have normal levels of CD55 and CD59 expression and hold promise as a potential source of HSCs for autologous transplantation to cure PNH patients.
Bibliografie:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23
ISSN:	0939-5555 1432-0584 1432-0584
DOI:	10.1007/s00277-016-2756-1