Hematopoietic stem cell transplantation for inborn errors of metabolism: A report from the Research Committee on Transplantation for Inborn Errors of Metabolism of the Japanese Ministry of Health, Labour and Welfare and the Working Group of the Japan Soci

2016 ◽  
Vol 20 (2) ◽  
pp. 203-214 ◽  
Author(s):  
Shunichi Kato ◽  
Hiromasa Yabe ◽  
Hiromitsu Takakura ◽  
Hideo Mugishima ◽  
Mika Ishige ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Working Group ◽  
Inborn Errors Of Metabolism ◽  
Research Committee ◽  
Inborn Errors ◽  
Hematopoietic Stem

scholarly journals β3-Adrenoreceptors as ROS Balancer in Hematopoietic Stem Cell Transplantation

2021 ◽  
Vol 22 (6) ◽  
pp. 2835
Author(s):  
Amada Pasha ◽  
Maura Calvani ◽  
Claudio Favre
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Therapeutic Potential ◽  
Redox Homeostasis ◽  
Primary Role ◽  
Inborn Errors ◽  
Hematopoietic Stem

In the last decades, the therapeutic potential of hematopoietic stem cell transplantation (HSCT) has acquired a primary role in the management of a broad spectrum of diseases including cancer, hematologic conditions, immune system dysregulations, and inborn errors of metabolism. The different types of HSCT, autologous and allogeneic, include risks of severe complications including acute and chronic graft-versus-host disease (GvHD) complications, hepatic veno-occlusive disease, lung injury, and infections. Despite being a dangerous procedure, it improved patient survival. Hence, its use was extended to treat autoimmune diseases, metabolic disorders, malignant infantile disorders, and hereditary skeletal dysplasia. HSCT is performed to restore or treat various congenital conditions in which immunologic functions are compromised, for instance, by chemo- and radiotherapy, and involves the administration of hematopoietic stem cells (HSCs) in patients with depleted or dysfunctional bone marrow (BM). Since HSCs biology is tightly regulated by oxidative stress (OS), the control of reactive oxygen species (ROS) levels is important to maintain their self-renewal capacity. In quiescent HSCs, low ROS levels are essential for stemness maintenance; however, physiological ROS levels promote HSC proliferation and differentiation. High ROS levels are mainly involved in short-term repopulation, whereas low ROS levels are associated with long-term repopulating ability. In this review, we aim summarize the current state of knowledge about the role of β3-adrenoreceptors (β3-ARs) in regulating HSCs redox homeostasis. β3-ARs play a major role in regulating stromal cell differentiation, and the antagonist SR59230A promotes differentiation of different progenitor cells in hematopoietic tumors, suggesting that β3-ARs agonism and antagonism could be exploited for clinical benefit.

scholarly journals Brazilian Consensus Guidelines for Hematopoietic Stem Cell Transplantation - Inborn errors of metabolismo

JBMTCT ◽  
2021 ◽  
Vol 2 (4) ◽  
pp. 126
Author(s):  
Alessandra Araujo Gomes ◽  
Adriana Mello Rodrigues ◽  
Juliana Folloni Fernandes ◽  
Liane Daudt ◽  
Carmem Bonfim
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Bone Marrow Transplantation ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Marrow Transplantation ◽  
Inborn Errors ◽  
Hematopoietic Stem

Hematopoietic stem cell transplantation (HSCT) has the potential to cure a significant proportion of patients with malignant and nonmalignant diseases. The main rationale for HSCT in inborn errors of metabolism (IEM) is based on correcting the decreases enzymes by the donor cells within and outside the intravascular compartment. In this article, Brazilian Group for Pediatric Bone Marrow Transplantation of the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy (SBTMO) provides a review of HSCT indications in IEM.

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