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Krabbe Translational Research Network (KTRN)

The third annual Krabbe Translation Research Network (KTRN) meeting was held March 6–8, 2013 in Coral Gables, Florida. Among the topics discussed were potential mechanisms underlying neuronal defects and motor dysfunction in Krabbe disease, and recent advances in neuroimaging technologies. Promising preliminary results were reported for reduced-intensity conditioning to improve outcomes after umbilical cord blood transplantation.

Researchers working in related fields were invited to share their insights regarding peripheral neuropathy, gene therapy in other leukodystrophies, and techniques for identifying modifying genetic factors, which may explain the wide phenotypic variation in Krabbe disease.

Currently under investigation in the labs of KTRN members are several approaches to gene therapy and combination therapy. In addition, a number of cellular products are being developed to augment transplantation, and several drugs with neuroprotective properties will soon be tested in mice.

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