Hematopoietic stem cell transplantation leads to biochemical and functional correction in two mouse models of acid ceramidase deficiency.

Autor: Rybova J; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, USA., Sundararajan T; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, USA., Kuchar L; Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic., Dlugi TA; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, USA., Ruzicka P; Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic., McKillop WM; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, USA., Medin JA; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, USA; Department of Biochemistry, Medical College of Wisconsin, Milwaukee, WI 53226, USA. Electronic address: jmedin@mcw.edu.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Oct 02; Vol. 32 (10), pp. 3402-3421. Date of Electronic Publication: 2024 Aug 05.

Novel anti-CD30/CD3 bispecific antibodies activate human T cells and mediate potent anti-tumor activity.

Autor: Faber ML; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., Oldham RAA; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States.; Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada., Thakur A; Department of Medicine, Division of Hematology/Oncology, University of Virginia Cancer Center, Charlottesville, VA, United States., Rademacher MJ; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., Kubicka E; Department of Medicine, Division of Hematology/Oncology, University of Virginia Cancer Center, Charlottesville, VA, United States., Dlugi TA; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., Gifford SA; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., McKillop WM; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., Schloemer NJ; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States., Lum LG; Department of Medicine, Division of Hematology/Oncology, University of Virginia Cancer Center, Charlottesville, VA, United States., Medin JA; Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, United States.; Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada.; Department of Biochemistry, MCW, Milwaukee, WI, United States.

Publikováno v: Frontiers in immunology [Front Immunol] 2023 Aug 14; Vol. 14, pp. 1225610. Date of Electronic Publication: 2023 Aug 14 (Print Publication: 2023).

Endocytosis of the thrombopoietin receptor Mpl regulates megakaryocyte and erythroid maturation in mice.

Autor: Eaton N; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States.; Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI, United States., Boyd EK; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States.; Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI, United States., Biswas R; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States., Lee-Sundlov MM; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States., Dlugi TA; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States., Ramsey HE; Division of Newborn Medicine, Boston Children's Hospital, Boston, MA, United States.; Department of Pediatrics, Harvard Medical School, Boston, MA, United States., Zheng S; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States., Burns RT; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States., Sola-Visner MC; Division of Newborn Medicine, Boston Children's Hospital, Boston, MA, United States.; Department of Pediatrics, Harvard Medical School, Boston, MA, United States., Hoffmeister KM; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States.; Departments of Medicine and Biochemistry, Medical College of Wisconsin, Milwaukee, WI, United States., Falet H; Translational Glycomics Center, Versiti Blood Research Institute, Milwaukee, WI, United States.; Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI, United States.

Publikováno v: Frontiers in oncology [Front Oncol] 2022 Aug 30; Vol. 12, pp. 959806. Date of Electronic Publication: 2022 Aug 30 (Print Publication: 2022).