Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders

Wagemaker G.

HUMAN GENE THERAPY, vol.25, no.10, pp.862-865, 2014 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 25 Issue: 10
  • Publication Date: 2014
  • Doi Number: 10.1089/hum.2014.102
  • Title of Journal : HUMAN GENE THERAPY
  • Page Numbers: pp.862-865


After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.