Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
暂无分享,去创建一个
Lei Zhang | Michael L Kaufman | David Gray | Pei-Qi Liu | Aaron R Cooper | Donald B Kohn | David E. Paschon | Lei Zhang | E. Rebar | P. Gregory | A. Reik | M. Holmes | G. Cost | M. Mendel | D. Kohn | A. Wilber | R. Hollis | Megan D Hoban | Xiaoyan Wang | Edward J Rebar | Matthew C Mendel | Philip D Gregory | Michael C Holmes | Alok V. Joglekar | Andreas Reik | Xiaoyan Wang | David E Paschon | Gregory J Cost | Zulema Romero | Michelle W L Ho | Alok V Joglekar | Michelle Ho | Dianne Lumaquin | Georgia R Lill | Fabrizia Urbinati | Shantha Senadheera | Allen Zhu | Andrew Wilber | Roger P Hollis | F. Urbinati | Dianne Lumaquin | Z. Romero | S. Senadheera | David Gray | Pei‐Qi Liu | A. Zhu | Pei‐Qi Liu | Pei‐Qi Liu
[1] D. Gordenin,et al. Chromosomal site-specific double-strand breaks are efficiently targeted for repair by oligonucleotides in yeast , 2003, Proceedings of the National Academy of Sciences of the United States of America.
[2] P. Malik,et al. Gene therapy for hemoglobinopathies: the state of the field and the future. , 2014, Hematology/oncology clinics of North America.
[3] Shondra M. Pruett-Miller,et al. Nuclease-mediated gene editing by homologous recombination of the human globin locus , 2013, Nucleic acids research.
[4] K. Sullivan,et al. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. , 2001, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.
[5] T. Delea,et al. Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States , 2008, American journal of hematology.
[6] Laurent Kiger,et al. Ex vivo generation of fully mature human red blood cells from hematopoietic stem cells , 2005, Nature Biotechnology.
[7] Shondra M Pruett-Miller,et al. High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases , 2011, Nature Methods.
[8] Morgan L. Maeder,et al. In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases , 2011, Stem cells.
[9] D. Greiner,et al. Creation of “Humanized” Mice to Study Human Immunity , 2008, Current protocols in immunology.
[10] K. Goncz,et al. Small fragment homologous replacement-mediated modification of genomic beta-globin sequences in human hematopoietic stem/progenitor cells. , 2006, Oligonucleotides.
[11] G. Serjeant. The natural history of sickle cell disease. , 1982, Cold Spring Harbor perspectives in medicine.
[12] Huimin Zhao,et al. Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs. , 2014, Biotechnology and bioengineering.
[13] Matthew Darlison,et al. Global epidemiology of haemoglobin disorders and derived service indicators. , 2008, Bulletin of the World Health Organization.
[14] M. Sadelain,et al. Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation. , 2014, Blood.
[15] M. van der Burg,et al. Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells , 2014, Nature.
[16] Xiao-Jin Yu,et al. Transient gene expression by nonintegrating lentiviral vectors. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[17] J. Stockman. Proof of principle for transfusion of in vitro–generated red blood cells , 2013 .
[18] P. Mali,et al. Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. , 2011, Blood.
[19] Michael L Kaufman,et al. β-globin gene transfer to human bone marrow for sickle cell disease. , 2013, The Journal of clinical investigation.
[20] P. Rouet,et al. Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease. , 1994, Molecular and cellular biology.
[21] Catherine J. Wu,et al. In mixed hematopoietic chimerism, the donor red cells win , 2011, Haematologica.
[22] Cage S. Johnson,et al. Outcome of Sickle Cell Anemia: A 4-Decade Observational Study of 1056 Patients , 2005, Medicine.
[23] M. Warr,et al. Hematopoietic stem cell quiescence promotes error-prone DNA repair and mutagenesis. , 2010, Cell stem cell.
[24] F. Mavilio,et al. Mechanisms of retroviral integration and mutagenesis. , 2013, Human gene therapy.
[25] Jeffrey C. Miller,et al. An unbiased genome-wide analysis of zinc-finger nuclease specificity , 2011, Nature Biotechnology.
[26] Vanessa Taupin,et al. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo , 2010, Nature Biotechnology.
[27] Jeffrey C. Miller,et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases , 2005, Nature.
[28] D. Kohn,et al. Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.
[29] Wei-Ting Hwang,et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. , 2014, The New England journal of medicine.
[30] Jérôme Larghero,et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia , 2010, Nature.