High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia-Reference-Cited by-同舟云学术

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia

Published:2014-01-30 Issue:5 Volume:123 Page:753-757
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Sullivan Spencer K.¹²,Mills Jason A.³,Koukouritaki Sevasti B.⁴⁵,Vo Karen K.⁶,Lyde Randolph B.⁶,Paluru Prasuna³,Zhao Guoha¹,Zhai Li¹,Sullivan Lisa M.³,Wang Yuhuan¹,Kishore Siddharth³,Gharaibeh Eyad Z.¹,Lambert Michele P.¹²,Wilcox David A.⁴⁵,French Deborah L.³,Poncz Mortimer¹²,Gadue Paul³

Affiliation:

1. Division of Hematology,

2. Department of Pediatrics, and

3. Department of Pathology and Laboratory Medicine, The Children’s Hospital of Philadelphia, Philadelphia, PA;

4. Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI;

5. Children’s Research Institute, Children’s Hospital of Wisconsin, Milwaukee, WI; and

6. Department of Pharmacology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

Abstract

Key Points When targeted to a single allele of the AAVS1 locus, the Gp1ba promoter drives a high level of expression specifically to megakaryocytes. Transgene rescue in iPSCs provides a model for the return of surface αIIbβ3 expression to near-normal levels in patients with type I GT.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/123/5/753/1379147/753.pdf

Reference22 articles.

1. Making Platelets Ex Vivo.;Lambert,2012

2. The GPIIb/IIIa (integrin alphaIIbbeta3) odyssey: a technology-driven saga of a receptor with twists, turns, and even a bend.;Coller;Blood,2008

3. Inherited platelet disorders.;Nurden;Haemophilia,2012

4. Natural history of platelet antibody formation against αIIbβ3 in a French cohort of Glanzmann thrombasthenia patients.;Fiore;Haemophilia,2012

5. Indication for allogeneic stem cell transplantation in Glanzmann’s thrombasthenia.;Wiegering;Hamostaseologie,2013

Cited by 56 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. p65 signaling dynamics drive the developmental progression of hematopoietic stem and progenitor cells through cell cycle regulation;Nature Communications;2024-09-06

2. Packaging of supplemented urokinase into alpha granules of in vitro–grown megakaryocytes for targeted nascent clot lysis;Blood Advances;2024-07-12

3. Glanzmann Thrombasthenia 10 Years Later: Progress Made and Future Directions;Seminars in Thrombosis and Hemostasis;2024-03-18

4. Modeling primitive and definitive erythropoiesis with induced pluripotent stem cells;Blood Advances;2024-03-15

5. Packaging of supplemented urokinase into naked alpha-granules ofin vitro-grown megakaryocytes for targeted therapeutic delivery;2023-12-05