Gene therapy for lympho-hematopoietic disorders

Curr Hematol Rep. 2005 Jul;4(4):305-9.

Abstract

Inherited lympho-hematopoietic disorders are considered to be some of the most amenable targets for development of gene therapy because of their defined molecular biology and pathophysiology and the potential for corrected cells to exhibit profound growth and survival advantages. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects.

MeSH terms

  • Genetic Therapy* / adverse effects
  • Genetic Therapy* / standards
  • Hematologic Diseases / therapy*
  • Hematologic Neoplasms / therapy*
  • Lymphatic Diseases / therapy*
  • Mutagenesis, Insertional / physiology
  • Protein Deficiency / therapy
  • Receptors, Cytokine / deficiency
  • Risk Factors
  • Severe Combined Immunodeficiency / therapy

Substances

  • Receptors, Cytokine