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Gene therapy a possibility for metachromatic leukodystrophy?

Metachromatic leukodystrophy (MLD) is an inherited disease that causes progressively more severe neurological defects that result in death early in life. Individuals with MLD have a genetic defect that means they lack a protein known as ARSA. There are currently no therapies for MLD, largely because the barrier between the blood supply and brain -- which tightly regulates the substances that can enter the brain -- provides an obstacle that has yet to be overcome. In a study appearing in the November issue of the Journal of Clinical Investigation, Alessandra Biffi and colleagues from the San Raffaele Scientific Institute in Italy, now show that the neurological defects in mice lacking ARSA can be corrected by treatment with hematopoietic stem progenitor cells (HSPCs) genetically modified to express high levels of ARSA. Importantly, ASRA was detected in the neurons of the treated mice and was derived from microglial cells, which differentiate from HSPCs. The successful correction of the protein defect in neurons and of the neurological defects led the authors to suggest that HSPC gene therapy might be efficacious for the treatment of individuals with MLD.


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Contact: Karen Honey
press_releases@the-jci.org
212-342-4159
Journal of Clinical Investigation
1-Nov-2006


Page: 1

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