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Target Of Novel Gene Therapy Pathway

by Fran Pollner

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NIDCR researchers have pioneered a new pathway to ameliorate chronic pain: gene transfer by recombinant adenoviral vector to deliver a ?-endorphin gene directly into the meningeal tissues surrounding the spinal cord.

After long trial and error (see "Commentary"), they came up with a magic vector-target combo that induces nonneuronal cells to bathe the cord in analgesic balm.

The minigene is constructed so that the connective tissue cells of the pia mater will secrete ?-endorphin into the cord and the cerebrospinal fluid (CSF). The strategy has relevance for neurodegenerative disorders and spinal cord injuries as well. Part of the Clinical CenterÍs new bench-to-bedside initiative, the research was done in collaboration with the University of Pennsylvania in Philadelphia.

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Photograph of Michael Iadarola
Michael Iadarola tells his research story in Catalyst "Commentary"

"We are totally pumped up. This approach is working, at least in the animal model," said Michael Iadarola, explaining his poster on "Viral Gene Transfer Approaches to Treatment for Chronic Pain" at the NIH Research Festival. Delivery of the ?-endorphin gene into the CSF, he said, resulted in the ratÍs failure to exhibit the typical reaction to pain that would have resulted from an inflamed paw.

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