Plerixafor+G-CSF–mobilized CD34+ cells represent an optimal graft source for thalassemia gene therapy-Reference-Cited by-同舟云学术

Plerixafor+G-CSF–mobilized CD34+ cells represent an optimal graft source for thalassemia gene therapy

Published:2015-07-30 Issue:5 Volume:126 Page:616-619
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Karponi Garyfalia¹²,Psatha Nikoletta¹,Lederer Carsten Werner³,Adair Jennifer Eileen⁴⁵,Zervou Fani¹,Zogas Nikolaos¹,Kleanthous Marina³,Tsatalas Constantinos²,Anagnostopoulos Achilles¹,Sadelain Michel⁶,Rivière Isabelle⁶⁷,Stamatoyannopoulos George⁵,Yannaki Evangelia¹

Affiliation:

1. Gene and Cell Therapy Center, Hematology Department-Bone Marrow Transplantation Unit, George Papanicolaou Hospital, Thessaloniki, Greece;

2. School of Medicine, Democritus University of Thrace, Alexandroupolis, Greece;

3. Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;

4. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;

5. Department of Medicine and Markey Molecular Medicine Center, University of Washington, Seattle, WA;

6. Center for Cell Engineering, Memorial Sloan-Kettering Cancer Center, New York, NY; and

7. Cell Therapy and Cell Engineering Facility, Memorial Sloan-Kettering Cancer Center, New York, NY

Abstract

Key Points Effective gene correction and long-term engraftment of human thalassemic CD34+ cells mobilized with different strategies. Plerixafor+G-CSF–mobilized CD34+ cells produce higher β-globin/VCN and superior early engraftment over single agent-mobilized cells.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/126/5/616/1391307/616.pdf

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4. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine.;Borgna-Pignatti;Haematologica,2004

5. Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy.;Sadelain;Hum Gene Ther,2007

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