Mucolipidosis IV (MLIV) is a devastating early childhood neurological disease characterized by progressive neurodegeneration, leading to severe impairments in muscle coordination, cognitive deficits and retinal degeneration that causes blindness.

There is no effective treatment for the condition, symptoms of which usually appear within a year of birth -- and whose rarity makes it a low priority for pharmaceutical companies.

But a possible first therapy has been discovered by biologist Craig Montell and his team of scientists at UC Santa Barbara. In a new study, researchers in Montell's lab found that bone marrow transplantation (BMT) significantly delayed the onset of motor deficits in an MLIV mouse model. Their findings appear in the journal Human Molecular Genetics.