Literature DB >> 15467825

Lenti in red: progress in gene therapy for human hemoglobinopathies.

Christof von Kalle1, Christopher Baum, David A Williams.   

Abstract

Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic beta-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.

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Year:  2004        PMID： 15467825      PMCID： PMC518672          DOI： 10.1172/JCI23132

Source DB:  PubMed          Journal:  J Clin Invest        ISSN： 0021-9738            Impact factor:   14.808


  21 in total

Review 1.  Locus control regions.

Authors:  Qiliang Li; Kenneth R Peterson; Xiangdong Fang; George Stamatoyannopoulos
Journal:  Blood       Date:  2002-11-01       Impact factor: 22.113

2.  HIV-1 integration in the human genome favors active genes and local hotspots.

Authors:  Astrid R W Schröder; Paul Shinn; Huaming Chen; Charles Berry; Joseph R Ecker; Frederic Bushman
Journal:  Cell       Date:  2002-08-23       Impact factor: 41.582

3.  Leukemic transformation of normal murine erythroid progenitors: v- and c-ErbB act through signaling pathways activated by the EpoR and c-Kit in stress erythropoiesis.

Authors:  M von Lindern; E M Deiner; H Dolznig; M Parren-Van Amelsvoort; M J Hayman; E W Mullner; H Beug
Journal:  Oncogene       Date:  2001-06-21       Impact factor: 9.867

4.  Correction of sickle cell disease in transgenic mouse models by gene therapy.

Authors:  R Pawliuk; K A Westerman; M E Fabry; E Payen; R Tighe; E E Bouhassira; S A Acharya; J Ellis; I M London; C J Eaves; R K Humphries; Y Beuzard; R L Nagel; P Leboulch
Journal:  Science       Date:  2001-12-14       Impact factor: 47.728

5.  Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin.

Authors:  C May; S Rivella; J Callegari; G Heller; K M Gaensler; L Luzzatto; M Sadelain
Journal:  Nature       Date:  2000-07-06       Impact factor: 49.962

6.  Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.

Authors:  Derek A Persons; Esther R Allay; Nobukuni Sawai; Phillip W Hargrove; Thomas P Brent; Hideki Hanawa; Arthur W Nienhuis; Brian P Sorrentino
Journal:  Blood       Date:  2003-03-27       Impact factor: 22.113

7.  Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

Authors:  Salima Hacein-Bey-Abina; Françoise Le Deist; Frédérique Carlier; Cécile Bouneaud; Christophe Hue; Jean-Pierre De Villartay; Adrian J Thrasher; Nicolas Wulffraat; Ricardo Sorensen; Sophie Dupuis-Girod; Alain Fischer; E Graham Davies; Wietse Kuis; Lilly Leiva; Marina Cavazzana-Calvo
Journal:  N Engl J Med       Date:  2002-04-18       Impact factor: 91.245

Review 8.  Side effects of retroviral gene transfer into hematopoietic stem cells.

Authors:  Christopher Baum; Jochen Düllmann; Zhixiong Li; Boris Fehse; Johann Meyer; David A Williams; Christof von Kalle
Journal:  Blood       Date:  2003-01-02       Impact factor: 22.113

9.  Transcription start regions in the human genome are favored targets for MLV integration.

Authors:  Xiaolin Wu; Yuan Li; Bruce Crise; Shawn M Burgess
Journal:  Science       Date:  2003-06-13       Impact factor: 47.728

10.  Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector.

Authors:  Geetha Puthenveetil; Jessica Scholes; Denysha Carbonell; Naveen Qureshi; Ping Xia; Licheng Zeng; Shulian Li; Ying Yu; Alan L Hiti; Jiing-Kuan Yee; Punam Malik
Journal:  Blood       Date:  2004-08-03       Impact factor: 22.113
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  6 in total

1.  A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.

Authors:  Ajay Perumbeti; Tomoyasu Higashimoto; Fabrizia Urbinati; Robert Franco; Herbert J Meiselman; David Witte; Punam Malik
Journal:  Blood       Date:  2009-05-27       Impact factor: 22.113

2.  The sea urchin sns5 insulator protects retroviral vectors from chromosomal position effects by maintaining active chromatin structure.

Authors:  Danilo D'Apolito; Elena Baiamonte; Mariella Bagliesi; Rosalba Di Marzo; Roberta Calzolari; Leda Ferro; Vito Franco; Giovanni Spinelli; Aurelio Maggio; Santina Acuto
Journal:  Mol Ther       Date:  2009-04-07       Impact factor: 11.454

3.  Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.

Authors:  Paritha I Arumugam; Tomoyasu Higashimoto; Fabrizia Urbinati; Ute Modlich; Shawna Nestheide; Ping Xia; Catherine Fox; Andrea Corsinotti; Christopher Baum; Punam Malik
Journal:  Mol Ther       Date:  2009-08-25       Impact factor: 11.454

4.  Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.

Authors:  Daren Wang; Wei Zhang; Theodosia A Kalfa; Gregory Grabowski; Stella Davies; Punam Malik; Dao Pan
Journal:  Proc Natl Acad Sci U S A       Date:  2009-11-10       Impact factor: 11.205

Review 5.  Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.

Authors:  Michel Sadelain; Alex Chang; Leszek Lisowski
Journal:  Mol Ther       Date:  2009-10-20       Impact factor: 11.454

6.  Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras.

Authors:  Eirini P Papapetrou; Damian Kovalovsky; Laurent Beloeil; Derek Sant'angelo; Michel Sadelain
Journal:  J Clin Invest       Date:  2008-12-01       Impact factor: 14.808
  6 in total

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