Genetic medicines: Treatment strategies for hereditary disorders

Timothy P. O'Connor, Ronald G. Crystal

Research output: Contribution to journalReview article

116 Citations (Scopus)


The treatment of the more than 1,800 known monogenic hereditary disorders will depend on the development of 'genetic medicines' - therapies that use the transfer of DNA and/or RNA to modify gene expression to correct or compensate for an abnormal phenotype. Strategies include the use of somatic stem cells, gene transfer, RNA modification and, in the future, embryonic stem cells. Despite the efficacy of these technologies in treating experimental models of hereditary disorders, applying them successfully in the clinic is a great challenge, which will only be overcome by expending considerable intellectual and economic resources, and by solving societal concerns about modifications of the human genetic repertoire.

Original languageEnglish
Pages (from-to)261-276
Number of pages16
JournalNature Reviews Genetics
Issue number4
Publication statusPublished - 1 Apr 2006


ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Genetics(clinical)

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