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This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.
Anemia, Sickle Cell
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Not yet recruiting
Published on BioPortfolio: 2019-09-23T04:51:51-0400
Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients Wit
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains C...
How safe and effective are CD34+ cell intracoronary injections for treating coronary endothelial dysfunction (CED)?
Primary objective: To assess the differences in the overall survival at 3 years of a CD34+ cell selection versus no selection of hematopoietic progenitor cells harvested during peripheral...
Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-Thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected periph...
Typically, the collection of blood cells for autologous stem cell transplant is done after the drugs granulocyte colony-stimulating factor (G-CSF) and plerixafor have been given to activat...
Cellular therapies using engineered T cells, haploidentical transplants, and autologous gene therapy are increasing. Specified CD3+ or high CD34+ doses are typically required for subsequent manufactur...
Cell damage inescapably occurs during both the freezing and the thawing graft processes for autologous hematopoietic stem cell (HSC) transplantation. To estimate HSC injury, a quality control is perfo...
Umbilical cord blood CD34+stem cells (UCB-CD34+) are clinically used in hematopoietic cell transplantation. However, there are limitations in the use of umbilical cord blood transplants due to the sma...
B-cell progenitors (hematogones) are markedly decreased or completely absent in the bone marrows of myelodysplastic syndromes (MDS), and the finding is considered as an important feature in MDS flow c...
Factors affecting the success of peripheral blood stem cell collection (SCC) in children are not well characterized. We reviewed 218 stem cell collections among 199 pediatric donors, of which 35 were ...
A member of the prominin family, AC133 Antigen is a 5-transmembrane antigen occurring as several isoforms produced by alternative splicing which are processed into mature forms. In humans, it is expressed as a subset of CD34 (bright) human hematopoietic stem cells and CD34 positive leukemias. Functionally, it is associated with roles in cell differentiation, proliferation, and apoptosis. Specifically, it regulates the organization of apical plasma membrane in epithelial cells, disk morphogenesis during early retinal development, MAPK and Akt signaling pathways, and in cholesterol metabolism.
Glycoproteins found on immature hematopoietic cells and endothelial cells. They are the only molecules to date whose expression within the blood system is restricted to a small number of progenitor cells in the bone marrow.
A fractionated cell extract that maintains a biological function. A subcellular fraction isolated by ultracentrifugation or other separation techniques must first be isolated so that a process can be studied free from all of the complex side reactions that occur in a cell. The cell-free system is therefore widely used in cell biology. (From Alberts et al., Molecular Biology of the Cell, 2d ed, p166)
Methods of implanting a CELL NUCLEUS from a donor cell into an enucleated acceptor cell. Often the nucleus of a somatic cell is transferred into a recipient OVUM or stem cell (STEM CELLS) with the nucleus removed. This technology may provide means to generate autologous diploid pluripotent cell for therapeutic cloning, and a model for studying NUCLEAR REPROGRAMMING in embryonic stem cells. Nuclear transfer was first accomplished with frog eggs (RANA PIPIENS) and reported in 1952.
Detergent-insoluble CELL MEMBRANE components. They are enriched in SPHINGOLIPIDS and CHOLESTEROL and clustered with glycosyl-phosphatidylinositol (GPI)-anchored proteins.