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

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  • Author(s): von Kalle C;von Kalle C; Baum C; Williams DA
  • Source:
    The Journal of clinical investigation [J Clin Invest] 2004 Oct; Vol. 114 (7), pp. 889-91.
  • Publication Type:
    Comment; Journal Article
  • Language:
    English
  • Additional Information
    • Source:
      Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print Cited Medium: Print ISSN: 0021-9738 (Print) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE
    • Publication Information:
      Publication: 1999- : Ann Arbor, MI : American Society for Clinical Investigation
      Original Publication: New Haven [etc.] American Society for Clinical Investigation.
    • Subject Terms:
      Genetic Therapy*; Hemoglobinopathies/*genetics ; Hemoglobinopathies/*therapy; Animals ; Genetic Vectors ; Globins/genetics ; Humans ; Mice ; Transgenes
    • 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.
    • Comments:
      Comment on: J Clin Invest. 2004 Oct;114(7):953-62. (PMID: 15467834)
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    • Accession Number:
      9004-22-2 (Globins)
    • Publication Date:
      Date Created: 20041007 Date Completed: 20041122 Latest Revision: 20181113
    • Publication Date:
      20240829
    • Accession Number:
      PMC518672
    • Accession Number:
      10.1172/JCI23132
    • Accession Number:
      15467825