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      Hematopoietic stem cell transplantation in its 60s: A platform for cellular therapies

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          Abstract

          Over the last 60 years, more than a million patients received hematopoietic cell transplantation. Having incorporated multiple changes in clinical practices, it remains a complex procedure facing a dual challenge: cure of the underlying disease and prevention of relapse while controlling potentially severe complications. Improved understanding of underlying biological processes resulted in the design of innovative therapies engineered from defined cell populations and testing of these therapies as addition or substitution at virtually every step of the procedure. This review provides an overview of these developments, many of them now applied outside the historical field of hematopoietic cell transplantation.

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          Acute Graft-versus-Host Disease — Biologic Process, Prevention, and Therapy

          Dan L Longo, Robert Zeiser, Bruce R. Blazar (2017)
          Article 0 comments Cited 457 times – based on 0 reviews     Review now
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            Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

            M Cavazzana-Calvo, S Hacein-Bey, G de Saint Basile … (2000)
            Severe combined immunodeficiency-X1 (SCID-X1) is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation. This block is caused by mutations of the gene encoding the gammac cytokine receptor subunit of interleukin-2, -4, -7, -9, and -15 receptors, which participates in the delivery of growth, survival, and differentiation signals to early lymphoid progenitors. After preclinical studies, a gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells. After a 10-month follow-up period, gammac transgene-expressing T and NK cells were detected in two patients. T, B, and NK cell counts and function, including antigen-specific responses, were comparable to those of age-matched controls. Thus, gene therapy was able to provide full correction of disease phenotype and, hence, clinical benefit.
            Article 0 comments Cited 302 times – based on 0 reviews     Review now
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              Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

              Marion G Ott, Manfred Schmidt, Kerstin Schwarzwaelder … (2006)
              Gene transfer into hematopoietic stem cells has been used successfully for correcting lymphoid but not myeloid immunodeficiencies. Here we report on two adults who received gene therapy after nonmyeloablative bone marrow conditioning for the treatment of X-linked chronic granulomatous disease (X-CGD), a primary immunodeficiency caused by a defect in the oxidative antimicrobial activity of phagocytes resulting from mutations in gp91(phox). We detected substantial gene transfer in both individuals' neutrophils that lead to a large number of functionally corrected phagocytes and notable clinical improvement. Large-scale retroviral integration site-distribution analysis showed activating insertions in MDS1-EVI1, PRDM16 or SETBP1 that had influenced regulation of long-term hematopoiesis by expanding gene-corrected myelopoiesis three- to four-fold in both individuals. Although insertional influences have probably reinforced the therapeutic efficacy in this trial, our results suggest that gene therapy in combination with bone marrow conditioning can be successfully used to treat inherited diseases affecting the myeloid compartment such as CGD.
              Article 0 comments Cited 265 times – based on 0 reviews     Review now
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                Author and article information

                Journal
                Title: Science Translational Medicine
                Abbreviated Title: Sci. Transl. Med.
                Publisher: American Association for the Advancement of Science (AAAS)
                ISSN (Print): 1946-6234
                ISSN (Electronic): 1946-6242
                Publication date Created: April 11 2018
                Publication date (Electronic): April 11 2018
                Publication date (Print): April 11 2018
                Volume: 10
                Issue: 436
                Page: eaap9630
                Article
                DOI: 10.1126/scitranslmed.aap9630
                PubMed ID: 29643233
                SO-VID: 1579b97d-0dfc-4fe3-971c-826946bdc88e
                Copyright © © 2018
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                http://www.sciencemag.org/about/science-licenses-journal-article-reuse

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