Related references
Note: Only part of the references are listed.AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage
Christopher Binny et al.
BLOOD (2012)
It's Time for Gene Therapy to Get Disruptive!
James M. Wilson
HUMAN GENE THERAPY (2012)
Codon optimization of human factor VIII cDNAs leads to high-level expression
Natalie J. Ward et al.
BLOOD (2011)
AAV Vector Biology in Primates: Finding the Missing Link?
Roland W. Herzog et al.
MOLECULAR THERAPY (2011)
Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins
Amit C. Nathwani et al.
MOLECULAR THERAPY (2011)
Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B
Amit C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome >= 8.2kb
Yi Lai et al.
MOLECULAR THERAPY (2010)
Characterization of Genome Integrity for Oversized Recombinant AAV Vector
Biao Dong et al.
MOLECULAR THERAPY (2010)
Effect of Genome Size on AAV Vector Packaging
Zhijian Wu et al.
MOLECULAR THERAPY (2010)
Enhanced Factor VIII Heavy Chain for Gene Therapy of Hemophilia A
Lingxia Chen et al.
MOLECULAR THERAPY (2009)
Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette
Hui Lu et al.
HUMAN GENE THERAPY (2008)
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
Mariacarmela Allocca et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Antioxidants reduce endoplasmic reticulum stress and improve protein secretion
Jyoti D. Malhotra et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Analysis of factor VIII mediated suppression of lentiviral vector titres
P. A. Radcliffe et al.
GENE THERAPY (2008)
Correction of murine hemophilia a and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors
Vincenzo Cerullo et al.
MOLECULAR THERAPY (2007)
The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene
Lingxia Chen et al.
MOLECULAR THERAPY (2007)
Efficacy, safety and tolerability of recombinant factor VIII (REFACTO®) in patients with haemophilia A:: interim data from a postmarketing surveillance study in Germany and Austria
H. Pollmann et al.
HAEMOPHILIA (2007)
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
Amit C. Nathwani et al.
BLOOD (2007)
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B
Haiyan Jiang et al.
MOLECULAR THERAPY (2006)
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
AC Nathwani et al.
BLOOD (2006)
Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models
AM Davidoff et al.
MOLECULAR THERAPY (2005)
Efficacy and inhibitor development in previously treated patients with haemophilia A switched to a B domain-deleted recombinant factor VIII
A Gringeri et al.
BRITISH JOURNAL OF HAEMATOLOGY (2004)
Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
AM Davidoff et al.
JOURNAL OF VIROLOGICAL METHODS (2004)
Bioengineering of coagulation factor VIR for improved secretion
HZ Miao et al.
BLOOD (2004)
Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion
JS Powell et al.
BLOOD (2003)
The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A
JM Lusher et al.
HAEMOPHILIA (2003)
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
GP Gao et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Expression of human factor VIII by splicing between dimerized AAV vectors
HJ Chao et al.
MOLECULAR THERAPY (2002)
Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
DA Roth et al.
NEW ENGLAND JOURNAL OF MEDICINE (2001)