Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.

Gene transfer into the hematopoietic stem cell has shown curative potential for a variety of hematological disorders. Primary immunodeficiency diseases have led to the way in this field of gene therapy as an example and a model. Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. Unfortunately, with the initial clear clinical benefits, the first serious complications of gene therapy have also occurred. In a significant number of patients treated using vectors based on murine gamma-retroviruses and carrying powerful viral enhancer elements, insertional oncogenesis events have resulted in acute leukemias that, in some cases, have had fatal outcomes. These serious adverse events have sparked a revision of the assessment of risks and benefits of integrating gene transfer for hematological diseases and prompted the development and application of new generations of viral vectors with recognized superior safety characteristics. This review summarizes the clinical experience of gene therapy for primary immunodeficiencies and discusses the likely avenues of progress in the future development of this expanding field of clinical investigations.
AuthorsFabio Candotti
JournalInternational journal of hematology (Int J Hematol) Vol. 99 Issue 4 Pg. 383-92 (Apr 2014) ISSN: 1865-3774 [Electronic] Japan
PMID24488786 (Publication Type: Journal Article, Review)
Chemical References
  • Adenosine Deaminase
  • Adenosine Deaminase (deficiency, genetics)
  • Agammaglobulinemia (genetics, therapy)
  • Animals
  • Clinical Trials as Topic
  • Gene Transfer Techniques
  • Genetic Therapy
  • Granulomatous Disease, Chronic (genetics, therapy)
  • Hematopoietic Stem Cell Transplantation
  • Hematopoietic Stem Cells (metabolism)
  • Humans
  • Immunologic Deficiency Syndromes (genetics, therapy)
  • Severe Combined Immunodeficiency (genetics, therapy)
  • Wiskott-Aldrich Syndrome (genetics, therapy)
  • X-Linked Combined Immunodeficiency Diseases (genetics, therapy)

Join CureHunter, for free Research Interface BASIC access!

Take advantage of free CureHunter research engine access to explore the best drug and treatment options for any disease. Find out why thousands of doctors, pharma researchers and patient activists around the world use CureHunter every day.
Realize the full power of the drug-disease research network!

Choose Username:
Verify Password: