In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model-Reference-Cited by-同舟云学术

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

Published:2017-08 Issue:8 Volume:25 Page:1843-1853
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Mattar Citra N.Z.,Gil-Farina Irene,Rosales Cecilia,Johana Nuryanti,Tan Yvonne Yi Wan,McIntosh Jenny,Kaeppel Christine,Waddington Simon N.,Biswas Arijit,Choolani Mahesh,Schmidt Manfred,Nathwani Amit C.,Chan Jerry K.Y.

Funder

Singapore’s Ministry of Health’s National Medical Research Council

ERC

NC3Rs

MRC

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference48 articles.

1. Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques;Mattar;Mol. Ther.,2011

2. A rare cause of intracranial hemorrhage: factor X deficiency;Citak;Pediatr. Emerg. Care,2001

3. Homozygosity for the Mediterranean a-thalassemic deletion (hemoglobin Barts hydrops fetalis);Al-Allawi;Ann. Saudi Med.,2010

4. Alpha-thalassemia: Hb H disease and Hb Barts hydrops fetalis;Chui;Ann. N Y Acad. Sci.,2005

5. Human fetal mesenchymal stem cells as vehicles for gene delivery;Chan;Stem Cells,2005

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