Hematopoietic stem cell gene therapy for the cure of blood diseases: primary immunodeficiencies

Cristina Cifaldi, Francesca Ferrua, Alessandro Aiuti, Caterina Cancrini

Research output: Contribution to journalReview article

Abstract

This report provides an overview of gene therapy use of stem cell for the treatment of primary immunodeficiencies (PIDs). Human stem cells are non-specialized cells, able to differentiate, starting from the embryo and for the whole duration of the life of each individual, into different cell types, through two types of cell division: symmetrical creating two stem cells and asymmetric differentiating to any cell lineage. These cells represented the ideal target for gene correction to guarantee production of engineered multi-lineage progeny. Their use in gene therapy led to the development of an effective treatment for PIDs, such as ADA deficiency, SCID-X1, WAS, CGD, and providing potential long-term clinical benefit for affected patients.

Original languageEnglish
Pages (from-to)755-764
Number of pages10
JournalRendiconti Lincei
Volume29
Issue number4
DOIs
Publication statusPublished - Dec 1 2018

Keywords

  • Adenosine deaminase deficiency
  • Chronic granulomatous disease
  • Gene therapy
  • Hematopoietic stem cells
  • Primary immunodeficiencies
  • RAGs defect
  • SCID-X1
  • Wiskott–Aldrich syndrome
  • XLP1 disease

ASJC Scopus subject areas

  • Environmental Science(all)
  • Agricultural and Biological Sciences(all)
  • Earth and Planetary Sciences(all)

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