Correction of Genetic Blood Defects by Gene Transfer

  • Marina Cavazzana-Calvo
  • Salima Hacein-Bey-Abina
  • Adrian J. Thrasher
  • Philippe Leboulch
  • Alain Fischer


Gene therapy has been proposed as an appealing tool for introducing a normal gene into affected hematopoietic stem cells to correct their inherited defect. Theoretically, in the absence of a related human leukocyte antigen identical donor, gene therapy could be an alternative given the accessibility and the information available on the hematopoietic stem cell biology. This chapter describes the progress and limits of the gene therapy approach applied to some genetic blood defects that appear to be good targets for this strategy.

Key Words

ADA Fanconi anemia gene therapy hematopoietic SCD SCID 


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Copyright information

© Humana Press Inc. 2006

Authors and Affiliations

  • Marina Cavazzana-Calvo
    • 1
  • Salima Hacein-Bey-Abina
    • 1
  • Adrian J. Thrasher
    • 2
  • Philippe Leboulch
    • 3
  • Alain Fischer
    • 4
  1. 1.Department of Biotherapy AP-HPHôpital NeckerParisFrance
  2. 2.Molecular Immunology UnitInstitute of Child HealthLondonUK
  3. 3.Harvard Medical School and Genetics DivisionBrigham and Women’s HospitalBoston
  4. 4.Unité d’Immunologie et d’Hématologie PédiatriquesHôpital NeckerParisFrance

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