Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.
Department of Internal Medicine, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
Shannon L
SL
Macauley
Mark S
MS
Sands
D000818:Animals; D002490:Central Nervous System / Q000201:enzymology*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000473:pathology* / Q000628:therapy*; D013108:Sphingomyelin Phosphodiesterase / Q000008:administration & dosage / Q000235:genetics
D013108:Sphingomyelin Phosphodiesterase