Genetic medicines: treatment strategies for hereditary disorders. Review uri icon

Overview

MeSH

  • Gene Transfer Techniques
  • Humans
  • RNA
  • Stem Cell Transplantation

MeSH Major

  • Genetic Diseases, Inborn
  • Genetic Therapy
  • Genetics, Medical

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.

publication date

  • April 2006

has subject area

  • Gene Transfer Techniques
  • Genetic Diseases, Inborn
  • Genetic Therapy
  • Genetics, Medical
  • Humans
  • RNA
  • Stem Cell Transplantation

Research

keywords

  • Journal Article
  • Review

Identity

Language

  • eng

Digital Object Identifier (DOI)

  • 10.1038/nrg1829

PubMed ID

  • 16543931

Additional Document Info

start page

  • 261

end page

  • 276

volume

  • 7

number

  • 4