Gene therapy: repairing haemoglobin disorders with ribozymes

Curr Biol. 1998 Sep 24;8(19):R696-8. doi: 10.1016/s0960-9822(98)70439-7.

Abstract

A ribozyme-mediated approach has made it possible to replace the region in beta globin mRNA containing the sickle-cell-anaemia mutation with a gamma-globin-encoding sequence. This is an interesting new way of correcting monogenic disease, but there are major problems to overcome before it could be applied in the clinic.

Publication types

  • Review

MeSH terms

  • Anemia, Sickle Cell / genetics
  • Anemia, Sickle Cell / therapy
  • Animals
  • Blood Viscosity
  • Cells, Cultured
  • Erythroid Precursor Cells / drug effects
  • Erythroid Precursor Cells / metabolism
  • Fetal Hemoglobin / biosynthesis
  • Fetal Hemoglobin / genetics
  • Genetic Therapy* / adverse effects
  • Globins / genetics*
  • Hemoglobinopathies / genetics
  • Hemoglobinopathies / therapy*
  • Humans
  • Mice
  • Mice, Transgenic
  • RNA, Catalytic / therapeutic use*
  • RNA, Messenger / drug effects*
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism

Substances

  • RNA, Catalytic
  • RNA, Messenger
  • Globins
  • Fetal Hemoglobin