Globin gene transfer as a potential treatment for the β‐thalassaemias and sickle cell disease

[1]  M. Sadelain.  Insertional oncogenesis in gene therapy: how much of a risk? , 2004, Gene Therapy.

[2]  N. Copeland,et al.  Gene Therapy Insertional Mutagenesis Insights , 2004, Science.

[3]  Michel Sadelain,et al.  Occurrence of leukaemia following gene therapy of X-linked SCID , 2003, Nature Reviews Cancer.

[4]  Cameron S. Osborne,et al.  LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 , 2003, Science.

[5]  S. Rivella,et al.  Globin gene transfer: a paradigm for transgene regulation and vector safety , 2003 .

[6]  K. Cornetta,et al.  American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

[7]  S. Rivella,et al.  A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. , 2003, Blood.

[8]  A. Nienhuis,et al.  The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number. , 2003, Blood.

[9]  Christof von Kalle,et al.  Side effects of retroviral gene transfer into hematopoietic stem cells. , 2003, Blood.

[10]  L. Ailles,et al.  Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. , 2002, Blood.

[11]  R. Nagel,et al.  Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[12]  S. Rivella,et al.  Therapeutic globin gene delivery using lentiviral vectors. , 2002, Current opinion in molecular therapeutics.

[13]  G. Stamatoyannopoulos,et al.  Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo. , 2002, Blood.

[14]  Takeshi Suzuki,et al.  New genes involved in cancer identified by retroviral tagging , 2002, Nature Genetics.

[15]  Anton Berns,et al.  High-throughput retroviral tagging to identify components of specific signaling pathways in cancer , 2002, Nature Genetics.

[16]  R. Nagel,et al.  Expression of a human beta-globin transgene in erythroid cells derived from retrovirally transduced transplantable human fetal liver and cord blood cells. , 2002, Blood.

[17]  J. Strouboulis,et al.  Stochastic patterns in globin gene expression are established prior to transcriptional activation and are clonally inherited. , 2002, Molecular cell.

[18]  G. Stamatoyannopoulos,et al.  Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[19]  C. von Kalle,et al.  Murine Leukemia Induced by Retroviral Gene Marking , 2002, Science.

[20]  R. A. Swank,et al.  Activation of the β-like globin genes in transgenic mice is dependent on the presence of the β-locus control region , 2002 .

[21]  M. Groudine,et al.  ChIPs of the β-globin locus: unraveling gene regulation within an active domain , 2002 .

[22]  P. Levings,et al.  The human β‐globin locus control region , 2002 .

[23]  S. Rivella,et al.  Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene , 2002 .

[24]  P. Mangeot,et al.  High levels of transduction of human dendritic cells with optimized SIV vectors. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[25]  Bryan R. Cullen,et al.  Recruitment of the Crm1 Nuclear Export Factor Is Sufficient To Induce Cytoplasmic Expression of Incompletely Spliced Human Immunodeficiency Virus mRNAs , 2002, Journal of Virology.

[26]  B. Cullen,et al.  Both ran and importins have the ability to function as nuclear mRNA export factors. , 2002, RNA.

[27]  L. Naldini,et al.  Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. , 2002, Human gene therapy.

[28]  S. Rivella,et al.  A novel murine model of Cooley’s anemia and its rescue by lentiviral mediated human ß-globin gene transfer , 2002 .

[29]  L. Naldini,et al.  Generation of HIV-1 derived lentiviral vectors. , 2002, Methods in enzymology.

[30]  M. Groudine,et al.  ChIPs of the beta-globin locus: unraveling gene regulation within an active domain. , 2002, Current opinion in genetics & development.

[31]  P. Levings,et al.  The human beta-globin locus control region. , 2002, European journal of biochemistry.

[32]  D. Trono,et al.  Lentiviral vectors for the gene therapy of lympho-hematological disorders. , 2002, Current topics in microbiology and immunology.

[33]  S. Rivella,et al.  Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. , 2002, Blood.

[34]  R. A. Swank,et al.  Activation of the beta-like globin genes in transgenic mice is dependent on the presence of the beta-locus control region. , 2002, Human molecular genetics.

[35]  R. Nagel,et al.  Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy , 2001, Science.

[36]  S. Goff.  Intracellular trafficking of retroviral genomes during the early phase of infection: viral exploitation of cellular pathways , 2001, The journal of gene medicine.

[37]  T. Somervaille.  Disorders of Hemoglobin: Genetics, Pathophysiology, and Clinical Management , 2001 .

[38]  M. Kumar,et al.  Systematic determination of the packaging limit of lentiviral vectors. , 2001, Human gene therapy.

[39]  R. Grady,et al.  Chelation therapy in β-thalassemia: An optimistic update☆ , 2001 .

[40]  D. Loukopoulos,et al.  Pharmacological induction of fetal hemoglobin in sickle cell disease and beta-thalassemia. , 2001, Seminars in hematology.

[41]  M. Sadelain,et al.  Toward gene therapy for disorders of globin synthesis. , 2001, Seminars in hematology.

[42]  D. Kohn.  Gene therapy for genetic haematological disorders and immunodeficiencies , 2001, Journal of internal medicine.

[43]  D. J. Weatherall,et al.  Phenotype—genotype relationships in monogenic disease: lessons from the thalassaemias , 2001, Nature Reviews Genetics.

[44]  R. Grady,et al.  Chelation therapy in beta-thalassemia: an optimistic update. , 2001, Seminars in hematology.

[45]  M. Sadelain,et al.  Lentivirus-transduced human monocyte-derived dendritic cells efficiently stimulate antigen-specific cytotoxic T lymphocytes. , 2001, Blood.

[46]  I. Meeus,et al.  In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction. , 2000, Blood cells, molecules & diseases.

[47]  D. Bodine,et al.  Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[48]  M. Sadelain,et al.  Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells , 2000, Current opinion in hematology.

[49]  D. Bodine,et al.  A Minimal Ankyrin Promoter Linked to a Human γ-Globin Gene Demonstrates Erythroid Specific Copy Number Dependent Expression with Minimal Position or Enhancer Dependence in Transgenic Mice* , 2000, The Journal of Biological Chemistry.

[50]  G. Stamatoyannopoulos,et al.  A chromatin insulator protects retrovirus vectors from chromosomal position effects. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[51]  Michel Sadelain,et al.  Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin , 2000, Nature.

[52]  J. Dick,et al.  Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[53]  S. Rivella,et al.  The cHS4 Insulator Increases the Probability of Retroviral Expression at Random Chromosomal Integration Sites , 2000, Journal of Virology.

[54]  I. London,et al.  Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human beta-globin in engrafted mice. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[55]  J. D. Engel,et al.  Looping, Linking, and Chromatin Activity New Insights into β-globin Locus Regulation , 2000, Cell.

[56]  S. Rivella,et al.  Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. , 2000, Nature.

[57]  M. Groudine,et al.  Looping versus linking: toward a model for long-distance gene activation. , 1999, Genes & development.

[58]  P. Brown,et al.  Transduction of Human Progenitor Hematopoietic Stem Cells by Human Immunodeficiency Virus Type 1-Based Vectors Is Cell Cycle Dependent , 1999, Journal of Virology.

[59]  G. Stamatoyannopoulos,et al.  Analysis of gamma-globin expression cassettes in retrovirus vectors. , 1999, Human gene therapy.

[60]  F. Grosveld.  Activation by locus control regions? , 1999, Current opinion in genetics & development.

[61]  M. Andreani,et al.  Bone marrow transplantation in adult thalassemic patients. , 1999, Blood.

[62]  B. Torbett,et al.  Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. , 1999, Science.

[63]  D. Higgs.  Do LCRs Open Chromatin Domains? , 1998, Cell.

[64]  Fred H. Gage,et al.  Development of a Self-Inactivating Lentivirus Vector , 1998, Journal of Virology.

[65]  E. Sariban,et al.  Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium , 1998, Bone Marrow Transplantation.

[66]  R. A. Swank,et al.  Fetal gene reactivation. , 1998, Current opinion in genetics & development.

[67]  E. Rubin,et al.  Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease. , 1997, Science.

[68]  Minoru Yoshida,et al.  CRM1 Is an Export Receptor for Leucine-Rich Nuclear Export Signals , 1997, Cell.

[69]  M. Sadelain.  GENETIC TREATMENT OF THE HAEMOGLOINOPATHIES: RECOMBINATIONS AND NEW COMBINATIONS , 1997, British journal of haematology.

[70]  F. Grosveld,et al.  Evaluation of β-globin gene therapy constructs in single-copy transgenic mice. , 1997 .

[71]  F. Grosveld,et al.  Evaluation of beta-globin gene therapy constructs in single copy transgenic mice. , 1997, Nucleic acids research.

[72]  P. Wong,et al.  Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region. , 1996, Blood.

[73]  Roger Y Tsien,et al.  Identification of a signal for rapid export of proteins from the nucleus , 1995, Cell.

[74]  Utz Fischer,et al.  The HIV-1 Rev Activation Domain is a nuclear export signal that accesses an export pathway used by specific cellular RNAs , 1995, Cell.

[75]  F. Grosveld,et al.  Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. , 1995, Proceedings of the National Academy of Sciences of the United States of America.

[76]  D. Tuan,et al.  Mutagenesis of retroviral vectors transducing human beta‐globin gene and beta‐globin locus control region derivatives results in stable transmission of an active transcriptional structure. , 1994, The EMBO journal.

[77]  G. Lucarelli,et al.  Bone marrow transplantation in the treatment of thalassemia. , 1994, Current opinion in hematology.

[78]  M. Emerman,et al.  Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus , 1994, Journal of virology.

[79]  T. Hoang,et al.  Sickle cell disease of transgenic SAD mice. , 1994, Blood.

[80]  I. Plavec,et al.  A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. , 1993, Blood.

[81]  George J. Dover,et al.  Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia [see comments] , 1992 .

[82]  Richard D Moore,et al.  Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia. , 1992, Blood.

[83]  Y. Kan,et al.  A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression. , 1992, Proceedings of the National Academy of Sciences of the United States of America.

[84]  W. C. Forrester,et al.  High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. , 1990, Proceedings of the National Academy of Sciences of the United States of America.

[85]  S. Karlsson,et al.  Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. , 1989, Proceedings of the National Academy of Sciences of the United States of America.

[86]  M. Bender,et al.  A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells , 1989, Molecular and cellular biology.

[87]  P. Goodfellow,et al.  A simple disease with no cure , 1989, Nature.

[88]  P. Goodfellow,et al.  Sickle cell anaemia. A simple disease with no cure. , 1989, Nature.

[89]  E. Beutler,et al.  The molecular basis of blood diseases. G. Stamatoyannopoulos, A.W. Nienhuis, P. Leder, P.W. Majerus. Philadelphia: W.B. Saunders Company, 1987, 747 pp $95.00 , 1988 .

[90]  R. Mulligan,et al.  Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells , 1988, Nature.

[91]  G. Stamatoyannopoulos,et al.  Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein. , 1987, Proceedings of the National Academy of Sciences of the United States of America.

[92]  R. Mulligan,et al.  Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector , 1987, Molecular and cellular biology.

[93]  S. Orkin,et al.  Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. , 1984, The Journal of clinical investigation.

[94]  D. Labie,et al.  Structural bases of the inhibitory effects of hemoglobin F and hemoglobin A2 on the polymerization of hemoglobin S. , 1979, Proceedings of the National Academy of Sciences of the United States of America.

[95]  L. Pauling,et al.  Sickle cell anemia a molecular disease. , 1949, Science.

[96]  T. Cooley,et al.  A series of cases of splenomegaly in children with anemia and peculiar bone changes , 1925 .

[97]  Tangvarasittichai Surapon.  Thalassemia Syndrome , 2022 .