A gene therapy approach to an incurable degenerative braindisease has shown promise in cultured cells derived frompatients with the disorder.

Reporting in the current Proceedings of the National Academyof Sciences, university researchers described replacing thedefective enzyme in Niemann-Pick disease. The degeneration iscaused by accumulation of lipid in neurons, but in culturedfibroblasts repaired by the therapy, the lipid content wasreduced to normal.

The repaired cells also were able to normally process the lipid,called sphingomyelin, reported researchers from HebrewUniversity in Israel, and Mount Sinai School of Medicine andSloan-Kettering Cancer Center in New York.

There is no effective therapy for the disease, which killschildren by age 3. A less-devastating form of the disease doesnot involve the nervous system, and individuals can surviveinto their third decade.

The researchers also reported that they have devised a way tosort out the successfully altered cells without the need for amarker gene to identify them from cells that fail to take in thetherapeutic gene.

-- Roberta Friedman, Ph.D. Special to BioWorld

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