Search Results - bone+marrow+transplants

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Gene Therapy for DOCK8 Deficiency (UCLA Case No. 2023-178)

UCLA researchers in the Department of Pediatrics Allergy and Immunology have developed a gene therapy method to express the full-length DOCK8 protein in cells, aimed at treating DOCK8 deficiency. These method allows for safe and effective DOCK8 expression in hematopoietic stem cells without the need for an HLA-matched donor, enabling patients to undergo...

Published: 2/18/2025 | Inventor(s): Caroline Kuo

Keywords(s): bone marrow, bone marrow transplants, DOCK8, DOCK8 deficiency, Gene Editing Systems, Gene Therapy, hematopoietic stem cells, Immune System, immunodeficiency, Immunology, intein splicing

Category(s): Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Life Science Research Tools > Plasmids/Vectors

Identification of a Factor That Rescues Dysregulated Endocytosis in Cultured Human Hematopoietic Stem Cells (UCLA Case No. 2023-051)

UCLA researchers in the Departments of Molecular, Cell & Developmental Biology have developed a novel method to enhance the function of hematopoietic stem cells (HSCs) to improve the transplantability of ex vivo expanded human HSCs in clinical settings. BACKGROUND: Hematopoietic stem cells (HSCs), present in blood and bone marrow, can form mature...

Published: 2/14/2025 | Inventor(s): Hanna Mikkola, Julia Aguade Gorgorio

Keywords(s): bone marrow, bone marrow transplants, CRISPR, endocytosis, engraftment, environmental sensing, hematopoietic stem cells, leukemia, MLLT3, MYCT1, transplantability

Category(s): Therapeutics > Hematology, Life Science Research Tools, Therapeutics > Stem Cells And Regenerative Medicine