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Gene Therapy of Genetic Diseases of Blood Cells

Stem Cells

  1. Gabriela Kuftinec,
  2. Jennifer Wherley,
  3. Donald B. Kohn

Published Online: 10 FEB 2013

DOI: 10.1002/3527600906.mcb.201200019

Reviews in Cell Biology and Molecular Medicine

Reviews in Cell Biology and Molecular Medicine

How to Cite

Kuftinec, G., Wherley, J. and Kohn, D. B. 2013. Gene Therapy of Genetic Diseases of Blood Cells. Reviews in Cell Biology and Molecular Medicine. .

Author Information

  1. University of California, Department of Microbiology, Immunology and Molecular Genetics, Los Angeles, CA, USA

Publication History

  1. Published Online: 10 FEB 2013

Abstract

The article contains sections titled:

  • Genetic Diseases of Blood Cells
  • Allogeneic HSCT as Treatment of Genetic Diseases of Blood Cells
  • Gene Therapy
  • Severe Combined Immune Deficiency (SCID)
    • Introduction
    • SCID due to ADA Deficiency
    • X-Linked SCID
  • Wiskott–Aldrich Syndrome
  • Chronic Granulomatous Disease
  • Hemoglobinopathies
  • Lysosomal Storage Diseases and Metabolic Diseases
  • Gene Correction by Homologous Recombination
  • Summary

Keywords:

  • Allogeneic;
  • Autologous;
  • Bone marrow transplant;
  • Gene therapy;
  • Graft-versus-host disease (GVHD);
  • Granulocyte colony-stimulating factor (G-CSF);
  • Hematopoietic stem cell (HSC);
  • Haplotype;
  • Hemoglobinopathies;
  • Homologous recombination;
  • Insertional oncogenesis;
  • LAM-PCR;
  • Lentivirus;
  • Myeloablation;
  • Primary immune deficiency;
  • Retroviral vector;
  • Retrovirus;
  • Syngeneic;
  • Transduction;
  • Zinc finger;
  • Zinc finger nuclease