Novel Strategy for Gene Therapy of Globoid Cell Leukodystrophy. 

Dr. Alessandra Biffi also reported on a novel strategy for gene therapy of globoid cell leukodystrophy.

Globoid leukodystrophy (GLD), also known as Krabbe Disease, is a genetic disease whose victims are unable to make galactocerebrosidase (GALC). The GALC enzyme helps to metabolize some of the lipid components of myelin. A gene therapy strategy for GLD based on hematopoietic stem cells (HSC) is under development. The gene transfer can be made by using a virus, called the lentivirus (LV). This has proved to be an efficient method to transfer the GALC gene to HSCs   The treated cells are able to express GALC at levels that are very high. However, this high level of GALC expression may also cause functional impairment, or even death, of the HSCs. It could be that the unnaturally high levels of GALC expressed by the treated HSCs are damaging to them. However, GALC doesn’t damage the descendents of HSCs. This suggests a new therapeutic strategy that is now being tested. The LV vector has been modified, so that when the GALC gene is transplanted to the HSCs, the GALC gene lies dormant until the HCS divides and differentiates. That is, the GALC gene is not expressed until it is safe to do so. This novel strategy avoids GALC damage in HSCs, while allowing sustained GALC expression in the progeny of the HSCs. Evaluation of this strategy is in progress.

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For additional information on this leukodystrophy research article, please contact:

Alessandra Biffi, MD

Source: The Myelin Project Annual Meeting - 2008

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