Methods and Compositions for Treating Disorders Caused By a Deficiency in a Gene Product of a CLN Gene
The present invention provides methods and compositions for treating a disorder associated with a deficiency in a gene product of a CLN gene in a subject (e.g., neuronal ceroid lipofuscinosis (NCL)), comprising administering to the subject an effective amount of a sphingolipid (e.g., galactosylceramide, ceramide, lysophosphatidic acid, sulfatide and any combination thereof), thereby treating the disorder in the subject.
US 20,100,152,123 [MORE INFO
BOUSTANY ROSE-MARY N [LB]
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