Medicine. Gene therapy that works

Science. 2013 Aug 23;341(6148):853-5. doi: 10.1126/science.1242551.
No abstract available

Publication types

  • Comment

MeSH terms

  • Cerebroside-Sulfatase / genetics*
  • Genetic Therapy / methods*
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Leukodystrophy, Metachromatic / therapy*
  • Male
  • Wiskott-Aldrich Syndrome / therapy*
  • Wiskott-Aldrich Syndrome Protein / genetics*

Substances

  • Wiskott-Aldrich Syndrome Protein
  • Cerebroside-Sulfatase