What is the role of gene therapy in the treatment of chronic granulomatous disease (CGD)?

Updated: Jul 29, 2019
  • Author: Roman J Nowicki, MD, PhD; Chief Editor: Dirk M Elston, MD  more...
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Answer

Gene therapy for hematopoietic cells (GT-HSC) represents an attractive alternative to HSCT as therapy for CGD patients without a matched donor. The observation that XR-CGD carriers in whom 10% or greater of normal neutrophils were healthy suggested that a minor functional correction of neutrophils would be sufficient to restore a normal phenotype in GT-HSC–treated patients. Nevertheless, GT-CGD is difficult to perform because corrected HSCs do not have a selective growth advantage compared with deficient cells, and, at the same time, a large number of cells needs to be corrected to ensure a good restoration of neutrophil activity. Thus, myeloablative conditioning is necessary to ensure an efficient engraftment of progenitor cells. [23, 37, 38, 39, 40]


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