Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency-Reference-Cited by-同舟云学术

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Published:2016-07-07 Issue:1 Volume:128 Page:45-54
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Cicalese Maria Pia¹²,Ferrua Francesca¹²³,Castagnaro Laura¹,Pajno Roberta²³,Barzaghi Federica¹²,Giannelli Stefania¹,Dionisio Francesca¹,Brigida Immacolata¹,Bonopane Marco¹,Casiraghi Miriam¹²,Tabucchi Antonella⁴,Carlucci Filippo⁴,Grunebaum Eyal⁵,Adeli Mehdi⁶,Bredius Robbert G.⁷,Puck Jennifer M.⁸,Stepensky Polina⁹,Tezcan Ilhan¹⁰,Rolfe Katie¹¹,De Boever Erika¹¹,Reinhardt Rickey R.¹¹,Appleby Jonathan¹¹,Ciceri Fabio³¹²,Roncarolo Maria Grazia¹³¹³¹⁴,Aiuti Alessandro¹²³^ORCID

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;

2. Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;

3. Vita-Salute San Raffaele University, Milan, Italy;

4. Department of Medical Biotechnology, University of Siena, Siena, Italy;

5. Division of Immunology and Allergy, The Hospital for Sick Children, Toronto, Canada;

6. Division of Immunology and Allergy, Hamad Medical Corporation, Doha, Qatar;

7. Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands;

8. Department of Pediatrics, University of California–San Francisco, San Francisco, CA;

9. Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Hospital, Jerusalem, Israel;

10. Department of Pediatrics, Section of Pediatric Immunology, Hacettepe University Faculty of Medicine, Ankara, Turkey;

11. GSK Research and Development, GlaxoSmithKline, Stevenage, United Kingdom, and Upper Merion, PA;

12. Hematology and Bone Marrow Transplant Unit, San Raffaele Scientific Institute, Milan, Italy; and

13. Department of Pediatrics Division of Stem Cell Transplantation and Regenerative Medicine, and

14. Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA

Abstract

Key PointsSurvival was 100% for 18 patients with ADA-SCID treated with genetically modified CD34+ cells (2.3-13.4 years follow up; median, 6.9 years). Long-term engraftment, immune reconstitution, and fewer severe infections were observed in 15 out of 18 patients without leukemic transformation.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/128/1/45/1395155/45.pdf

Reference58 articles.

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4. Brief report: hepatic dysfunction as a complication of adenosine deaminase deficiency.;Bollinger;N Engl J Med,1996

5. Primary immunodeficiencies in Switzerland: first report of the national registry in adults and children.;Ryser;J Clin Immunol,1988

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