Globin Gene Transfer for Therapy of Sickle Cell Anemia

Sadelain, Michel

Abstract

Funding Agency

• Agency: National Institute of Health (NIH)
• Institute: National Heart, Lung, and Blood Institute (NHLBI)
• Type: Research Project (R01)
• Project #: 1R01HL057612-01
• Application #: 2030848
• Study Section: Special Emphasis Panel (ZHL1-CSR-B (S1))
• Project Start: 1996-09-30
• Project End: 1999-08-31
• Budget Start: 1996-09-30
• Budget End: 1997-08-31
• Support Year: 1
• Fiscal Year: 1996

Institution

• Name: Sloan-Kettering Institute for Cancer Research
• DUNS #: 064931884
• City: New York
• State: NY
• Country: United States
• Zip Code: 10065

Publications

Papapetrou, Eirini P; Korkola, James E; Sadelain, Michel (2010) A genetic strategy for single and combinatorial analysis of miRNA function in mammalian hematopoietic stem cells. Stem Cells 28:287-96

Papapetrou, Eirini P; Kovalovsky, Damian; Beloeil, Laurent et al. (2009) Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras. J Clin Invest 119:157-68

Chang, Alex H; Stephan, Matthias T; Lisowski, Leszek et al. (2008) Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice. Mol Ther 16:1745-52

Lisowski, Leszek; Sadelain, Michel (2007) Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice. Blood 110:4175-8

Budak-Alpdogan, Tulin; Przybylowski, Mark; Gonen, Mithat et al. (2006) Functional assessment of the engraftment potential of gammaretrovirus-modified CD34+ cells, using a short serum-free transduction protocol. Hum Gene Ther 17:780-94

Chang, Alex H; Stephan, Matthias T; Sadelain, Michel (2006) Stem cell-derived erythroid cells mediate long-term systemic protein delivery. Nat Biotechnol 24:1017-21

Samakoglu, Selda; Lisowski, Leszek; Budak-Alpdogan, Tulin et al. (2006) A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol 24:89-94

Tabar, Viviane; Panagiotakos, Georgia; Greenberg, Edward D et al. (2005) Migration and differentiation of neural precursors derived from human embryonic stem cells in the rat brain. Nat Biotechnol 23:601-6

Sadelain, Michel; Lisowski, Leszek; Samakoglu, Selda et al. (2005) Progress toward the genetic treatment of the beta-thalassemias. Ann N Y Acad Sci 1054:78-91

Sadelain, Michel; Rivella, Stefano; Lisowski, Leszek et al. (2004) Globin gene transfer for treatment of the beta-thalassemias and sickle cell disease. Best Pract Res Clin Haematol 17:517-34