Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier

Proc Natl Acad Sci U S A. 2002 Apr 30;99(9):5760-2. doi: 10.1073/pnas.102175599.
No abstract available

Publication types

  • Comment
  • Review

MeSH terms

  • Animals
  • Blood-Brain Barrier*
  • Central Nervous System Diseases / therapy
  • Genetic Therapy / methods*
  • Humans
  • Lysosomal Storage Diseases / genetics*
  • Lysosomal Storage Diseases / therapy*
  • Lysosomes / metabolism
  • Mice
  • Mucopolysaccharidosis VII / therapy