New therapeutic approaches to mendelian disorders

N Engl J Med. 2010 Aug 26;363(9):852-63. doi: 10.1056/NEJMra0907180.
No abstract available

Publication types

  • Review

MeSH terms

  • Animals
  • Enzyme Replacement Therapy*
  • Gene Expression
  • Genetic Diseases, Inborn / therapy*
  • Genetic Therapy / methods*
  • Humans
  • Marfan Syndrome / genetics
  • Marfan Syndrome / therapy
  • Mutation
  • Progeria / genetics
  • Progeria / therapy
  • Proteins / genetics
  • RNA Precursors / metabolism
  • RNA Splicing

Substances

  • Proteins
  • RNA Precursors