Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells-Reference-Cited by-同舟云学术

Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells

Published:2014-01-16 Issue:3 Volume:123 Page:395-403
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Shi Qizhen¹²³⁴,Kuether Erin L.¹²³,Chen Yingyu²⁵,Schroeder Jocelyn A.¹²³,Fahs Scot A.²,Montgomery Robert R.¹²³⁴

Affiliation:

1. Department of Pediatrics, Medical College of Wisconsin,

2. Blood Research Institute, BloodCenter of Wisconsin,

3. Children’s Research Institute, Children’s Hospital of Wisconsin,

4. Midwest Athletes Against Childhood Cancer Fund Research Center, Milwaukee, WI; and

5. Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China

Abstract

Key Points Platelet-specific lentiviral gene delivery to human hematopoietic stem cells can efficiently introduce FVIII expression in human platelets. Human platelet–derived FVIII can ameliorate the hemophilic phenotype in an immunocompromised hemophilia A mouse model.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/123/3/395/1377263/395.pdf

Reference46 articles.

1. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation.;Hay;Br J Haematol,2006

2. Treatment characteristics and the risk of inhibitor development: a multicenter cohort study among previously untreated patients with severe hemophilia A.;Gouw;J Thromb Haemost,2007

3. Current options and new developments in the treatment of haemophilia.;Wong;Drugs,2011

5. Parallel use of by-passing agents in haemophilia with inhibitors: a critical review.;Ingerslev;Br J Haematol,2011

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