Therapeutic benefits in thalassemic mice transplanted with long-term−cultured bone marrow cells

• Published in: Experimental hematology Vol. 39; no. 3; pp. 375 - 383.e4
• Main Authors: Hatada, Seigo, Walton, William, Hatada, Tomoko, Wofford, Anne, Fox, Raymond, Liu, Naiyou, Lill, Michael C, Fair, Jeffery H, Kirby, Suzanne L, Smithies, Oliver
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier Inc 01.03.2011
• Subjects: Advanced Basic Science; Animals; Bone Marrow Cells - metabolism; Bone Marrow Transplantation; Cell Culture Techniques; Cells, Cultured; Coculture Techniques; Female; Gene Expression Regulation; Hematology, Oncology and Palliative Medicine; Male; Mice; Mice, Transgenic; Thalassemia - metabolism; Thalassemia - therapy; Time Factors; Transplantation, Autologous

Objective Autologous bone marrow (BM) cells with a faulty gene corrected by gene targeting could provide a powerful therapeutic option for patients with genetic blood diseases. Achieving this goal is hindered by the low abundance of therapeutically useful BM cells and the difficulty maintaining them...