Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation-Reference-Cited by-同舟云学术

Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation

Published:2014-03-06 Issue:10 Volume:123 Page:1483-1486
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Boulad Farid¹²,Wang Xiuyan¹³⁴,Qu Jinrong⁴,Taylor Clare⁴,Ferro Leda⁴,Karponi Garyfalia⁴,Bartido Shirley⁴,Giardina Patricia⁵,Heller Glenn⁶,Prockop Susan E.²,Maggio Aurelio⁷,Sadelain Michel¹³,Rivière Isabelle¹³⁴

Affiliation:

1. Center for Cell Engineering,

2. Bone Marrow Transplant Service, Department of Pediatrics,

3. Molecular Pharmacology and Chemistry Program, and

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

5. Department of Pediatrics, New York-Presbyterian Hospital-Weill Cornell Medical College, New York, NY;

6. Department of Epidemiology & Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, NY; and

7. Ospedale V. Cervello, Palermo, Italy

Abstract

Key Points Safe mobilization of CD34+ cells in adults with β-thalassemia and effective transduction with a globin vector under cGMP conditions. Stable vector copy number and β-globin expression in BFU-Es derived from engrafted CD34+ HPCs 6 months post-transplant in NSG mice.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/123/10/1483/1374618/1483.pdf

Reference25 articles.

1. Hematopoietic Stem-Cell Transplantation for the Inherited Bone Marrow Failure Syndromes

Cited by 62 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Gene Therapy for Hemoglobinopathies;Human Gene Therapy;2023-09-01

2. Biotechnological advances in gene therapy of hematopoietic stem cells: Systematic review and meta-analysis;Human Gene Therapy;2023-08-25

3. 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia;HemaSphere;2022-07-29

4. Globin vector regulatory elements are active in early hematopoietic progenitor cells;Molecular Therapy;2022-06

5. Beta‐thalassemia and the advent of new interventions beyond transfusion and iron chelation;British Journal of Clinical Pharmacology;2022-04-18