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

C Cifaldi, F Ferrua, A Aiuti, C Cancrini

Research output: Contribution to journalArticlepeer-review


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. © 2018, Accademia Nazionale dei Lincei.
Original languageEnglish
Pages (from-to)755-764
Number of pages10
JournalRendiconti Lincei
Issue number4
Publication statusPublished - 2018


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