Progress and prospects: gene therapy for inherited immunodeficiencies.

Haematopoietic stem cell transplantation (HSCT) is now widely used to treat primary immunodeficiencies (PID). For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. The success of gene therapy can be attributed, in part, to the selective advantage offered to gene-corrected cells, the avoidance of graft-versus-host disease and to the use of pre-conditioning in patients with chemotherapy to facilitate engraftment of corrected cells. Adverse events have been encountered and this has led to detailed characterization of retroviral vector integration profiles. A new generation of self-inactivating retroviral and lentiviral vectors have been designed to address these safety concerns, and are at an advanced stage of preparation for the next phase of clinical testing.
AuthorsW Qasim, H B Gaspar, A J Thrasher
JournalGene therapy (Gene Ther) Vol. 16 Issue 11 Pg. 1285-91 (Nov 2009) ISSN: 1476-5462 [Electronic] England
PMID19776764 (Publication Type: Journal Article, Review)
  • Animals
  • Gene Silencing
  • Genetic Therapy (methods, trends)
  • Humans
  • Immunologic Deficiency Syndromes (therapy)
  • Male
  • Mutagenesis, Insertional
  • Severe Combined Immunodeficiency (therapy)
  • Wiskott-Aldrich Syndrome (therapy)

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