Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier.
Sly
William S
WS
Edward A. Doisy Department of Biochemistry, Saint Louis University School of Medicine, 1402 South Grand Boulevard, St. Louis, MO 63104, USA. slyws@slu.edu
Vogler
Carole
C
D000818:Animals; D001812:Blood-Brain Barrier; D002493:Central Nervous System Diseases / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000235:genetics* / Q000628:therapy*; D008247:Lysosomes / Q000378:metabolism; D051379:Mice; D016538:Mucopolysaccharidosis VII / Q000628:therapy