adrenoleukodystrophy   2

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy — NEJM
> A total of 17 boys received Lenti-D gene therapy. At the time of the interim analysis, the median follow-up was 29.4 months (range, 21.6 to 42.0). All the patients had gene-marked cells after engraftment, with no evidence of preferential integration near known oncogenes or clonal outgrowth. Measurable ALD protein was observed in all the patients. No treatment-related death or graft-versus-host disease had been reported; 15 of the 17 patients (88%) were alive and free of major functional disability, with minimal clinical symptoms. One patient, who had had rapid neurologic deterioration, had died from disease progression. Another patient, who had had evidence of disease progression on MRI, had withdrawn from the study to undergo allogeneic stem-cell transplantation and later died from transplantation-related complications.
adrenoleukodystrophy  MRI  neurology  gene_therapy 
october 2017 by porejide

related tags

gene_therapy  mri  neurology 

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