Loading...
Gene therapy has been on a roller coaster — first hailed for its enormous potential then trashed because of several high-profile disasters. During the past few years, however, several successes have been reported. In a new article, researchers describe another apparent success.
X-linked adrenoleukodystrophy (ALD) is a fatal demyelinating disease of the central nervous system that is caused by a defect in a single gene called ABCD. ALD can be treated by allogeneic hematopoietic stem cell therapy; the hematopoietic stem cells become myelin-producing oligodendrocytes. However, the donor cells face potential immune rejection.
Two boys with ALD had no available stem cell donors. White cells that were enriched for their own hematopoietic stem cells…