Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

Frank J.T. Staal, Alessandro Aiuti, Marina Cavazzana

Research output: Contribution to journalReview articlepeer-review

Abstract

Gene therapy using patient's own stem cells is rapidly becoming an alternative to allogeneic stem cell transplantation, especially when suitably compatible donors cannot be found. The advent of efficient virus-based methods for delivering therapeutic genes has enabled the development of genetic medicines for inherited disorders of the immune system, hemoglobinopathies, and a number of devastating metabolic diseases. Here, we briefly review the state of the art in the field, including gene editing approaches. A growing number of pediatric diseases can be successfully cured by hematopoietic stem-cell-based gene therapy.

Original languageEnglish
Article number443
JournalFrontiers in Pediatrics
Volume7
DOIs
Publication statusPublished - Oct 31 2019

Keywords

  • clinical trial
  • curative treatment
  • gene editing
  • gene therapy
  • lysosomal storage disorder
  • SCID
  • sickle cell disease
  • thalassemia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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