Systematic ranking of inborn errors of metabolism as targets for gene therapy

Mol Genet Metab. 2010 Jul;100(3):215-8. doi: 10.1016/j.ymgme.2010.04.011.
No abstract available

MeSH terms

  • Animals
  • Genetic Therapy*
  • Humans
  • Infant, Newborn
  • Metabolism, Inborn Errors / classification*
  • Metabolism, Inborn Errors / therapy*
  • Neonatal Screening
  • Translational Research, Biomedical