Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.

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From: The New England Journal of Medicine(Vol. 385, Issue 21)
Publisher: Massachusetts Medical Society
Document Type: Report
Length: 34 words

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Abstract :

The efficacy of allogeneic hematopoietic stem-cell transplantation for Hurler syndrome is evaluated. Patients who received autologous hematopoietic stem and progenitor cells had extensive metabolic correction in the peripheral tissues and the central nervous system.

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Gale Document Number: GALE|A687383612