Recent advances in approach to treatment of genetic disorders: clinicians perspective

Indian Pediatr. 2007 May;44(5):361-9.

Abstract

There is no cure for most of the genetic disorders. The only option in most situations is prevention by counseling and prenatal diagnosis. However, over a decade, with the completion of the human genome project and other advances there is better understanding of pathogenesis, improvement in diagnostic strategies and various treatment avenues are opening up for these disorders. The aim of this article is to make the pediatricians aware of the approaches to treatment of common genetic disorders and recent available therapeutic interventions.

Publication types

  • Review

MeSH terms

  • Enzyme Activators / therapeutic use*
  • Genetic Diseases, Inborn / therapy*
  • Genetic Therapy*
  • Humans
  • Stem Cell Transplantation*

Substances

  • Enzyme Activators