Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially ...
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Eltrombopag effect on the engraftment efficiency of human hematopoietic stem cells in a NOD/SCID xenotransplant model. Methods: Mice were treated with filgrastim (Neupogen) (FG) with or without ...
The recent regulatory approval of the first hematopoietic stem and progenitor cell gene therapy (HSPC-GT) signals the start of a new era for gene therapy and highlights the potential contribution by ...
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