Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia-Reference-Cited by-同舟云学术

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

Published:2019-01-21 Issue:2 Volume:25 Page:234-241
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Marktel Sarah,Scaramuzza Samantha,Cicalese Maria Pia,Giglio Fabio,Galimberti Stefania,Lidonnici Maria Rosa,Calbi Valeria,Assanelli Andrea,Bernardo Maria Ester,Rossi Claudia,Calabria Andrea^ORCID,Milani Raffaella,Gattillo Salvatore,Benedicenti Fabrizio,Spinozzi Giulio,Aprile Annamaria,Bergami Alessandra,Casiraghi Miriam,Consiglieri Giulia,Masera Nicoletta,D’Angelo Emanuela,Mirra Nadia,Origa Raffaella,Tartaglione Immacolata,Perrotta Silverio,Winter Robert,Coppola Milena,Viarengo Gianluca,Santoleri Luca,Graziadei Giovanna,Gabaldo Michela,Valsecchi Maria Grazia,Montini Eugenio,Naldini Luigi,Cappellini Maria Domenica,Ciceri Fabio,Aiuti Alessandro,Ferrari Giuliana^ORCID

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/s41591-018-0301-6.pdf

Reference36 articles.

1. Taher, A. T., Weatherall, D. J. & Cappellini, M. D. Thalassaemia. Lancet 391, 155–167 (2018).

2. Baronciani, D. et al. Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010. Bone. Marrow. Transplant. 51, 536–541 (2016).

3. Naldini, L. Ex vivo gene transfer and correction for cell-based therapies. Nat. Rev. Genet. 12, 301–315 (2011).

4. Modell, B. & Darlison, M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull. World Health Organ. 86, 480–487 (2008).

5. Cavazzana-Calvo, M. et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318–322 (2010).

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