Genetic medicines

Treatment strategies for hereditary disorders

Timothy P. O'Connor, Ronald Crystal

Research output: Contribution to journalReview article

114 Citations (Scopus)

Abstract

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
Volume7
Issue number4
DOIs
Publication statusPublished - 1 Apr 2006
Externally publishedYes

Fingerprint

Adult Stem Cells
Medical Genetics
Embryonic Stem Cells
Transfer RNA
Genetic Therapy
Theoretical Models
Economics
RNA
Technology
Phenotype
Gene Expression
DNA
Genes
Therapeutics

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Genetics(clinical)

Cite this

Genetic medicines : Treatment strategies for hereditary disorders. / O'Connor, Timothy P.; Crystal, Ronald.

In: Nature Reviews Genetics, Vol. 7, No. 4, 01.04.2006, p. 261-276.

Research output: Contribution to journalReview article

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