Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII-Reference-Cited by-同舟云学术

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

Published:2016-02-04 Issue:5 Volume:127 Page:565-571
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Marcos-Contreras Oscar A.¹,Smith Shannon M.¹,Bellinger Dwight A.²,Raymer Robin A.²,Merricks Elizabeth²,Faella Armida¹,Pavani Giulia¹,Zhou Shangzhen¹,Nichols Timothy C.²³,High Katherine A.¹⁴⁵,Margaritis Paris¹⁵⁶

Affiliation:

1. Department of Pediatrics, The Children’s Hospital of Philadelphia, Philadelphia, PA;

2. Department of Pathology and Laboratory Medicine and

3. Department of Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, NC;

4. Howard Hughes Medical Institute and

5. Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA; and

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

Abstract

Key Points Dogs with an FVII G96E mutation (FVII-G96E) represent the most common human FVII mutation type and are ideal for testing new therapies. cFVII gene delivery in FVII-G96E dogs via AAV at a dose effective in humans showed stable and clinically therapeutic FVII expression.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/127/5/565/1394890/565.pdf

Reference38 articles.

1. Recessively inherited coagulation disorders.;Mannucci;Blood,2004

2. Factor VII deficiency.;Perry;Br J Haematol,2002

3. Major differences in bleeding symptoms between factor VII deficiency and hemophilia B.;Bernardi;J Thromb Haemost,2009

4. Long-term prophylaxis in severe factor VII deficiency.;Siboni;Haemophilia,2015

5. Factor VII deficiency.;Mariani;Semin Thromb Hemost,2009

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