Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
نویسندگان
چکیده
منابع مشابه
Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome).
Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated...
متن کاملHEMATOPOIESIS AND STEM CELLS Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
1Division of Hematology-Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of MN, Minneapolis, MN, and 2Stem Cell Transplantation Program, Division of Pediatric Hematology Oncology, Manton Center for Orphan Disease Research, Children’s Hospital Boston, and Dana-Farber Cancer Institute; Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical S...
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BACKGROUND Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for young Hurler patients. Despite halting of neurocognitive decline and improvement of life expectancy, the beneficial effect on the skeletal system is limited. As orthopedic complications are one of the most disabling factors following HSCT, this points to the need for new treatment strategies. The study summ...
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Purpose: Differentiation of Mouse embryonicstem cells into Hematopoietic cells. Materials and Methods: In this study, we used EB formation system for Hematopoietic differentiation of mouse embryonic stem cell (Royan B1) in suspension culture. EBs cultured in medium with Hematopoietic inducer cytokines (SCF, TPO, GMCSF, IL3, Flt3 and EPO) .presence of hematopoietic differentiated cell assessed ...
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ژورنال
عنوان ژورنال: Nature Communications
سال: 2019
ISSN: 2041-1723
DOI: 10.1038/s41467-019-11962-8