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Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Blood, 2006-11, Vol.108 (10), p.3321-3328

2006

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Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Blood, 2006-03, Vol.107 (5), p.1810-1817

2006

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Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Blood, 2010-06, Vol.115 (23), p.4678-4688

2010

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Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Blood, 2006-07, Vol.108 (1), p.107-115

2006

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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

The New England journal of medicine, 2017-12, Vol.377 (23), p.2215-2227

2017

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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

Nature medicine, 1999-01, Vol.5 (1), p.56-63

1999

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Viral vector-mediated RNA interference

Current opinion in pharmacology, 2010-10, Vol.10 (5), p.534-542

2010

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Preclinical Evaluation of An Anti-HCV miRNA Cluster for Treatment of HCV Infection

Molecular therapy, 2013-03, Vol.21 (3), p.588-601

2013

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Blood, 2003-04, Vol.101 (8), p.2963-2972

2003

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Posttranslational modifications of recombinant myotube-synthesized human factor IX

Blood, 2001-01, Vol.97 (1), p.130-138

2001

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Inhibition of hepatitis C virus replication using adeno‐associated virus vector delivery of an exogenous anti–hepatitis C virus microrna cluster

Hepatology (Baltimore, Md.), 2010-12, Vol.52 (6), p.1877-1887

2010

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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Nature genetics, 2000-03, Vol.24 (3), p.257-261

2000

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Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Blood, 2005-05, Vol.105 (9), p.3458-3464

2005

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Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

Blood, 2004-01, Vol.103 (1), p.85-92

2004

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Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

Journal of Virology, 1999-07, Vol.73 (7), p.5438-5447

1999

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Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

Human gene therapy, 2002-07, Vol.13 (11), p.1281-1291

2002

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Inadvertent Germline Transmission of AAV2 Vector: Findings in a Rabbit Model Correlate with Those in a Human Clinical Trial

Molecular therapy, 2006-06, Vol.13 (6), p.1064-1073

2006

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Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

Blood, 2003-09, Vol.102 (6), p.2031-2037

2003

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Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Blood, 2003-12, Vol.102 (12), p.3919-3926

2003

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Preclinical Gene Therapy Studies for Hemophilia Using Adeno-Associated Virus (AAV) Vectors

Seminars in thrombosis and hemostasis, 2004-04, Vol.30 (2), p.161-171

Couto, Linda B

2004

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Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

Blood, 2000-04, Vol.95 (8), p.2536-2542

2000

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Modified infusion procedures affect recombinant adeno- associated virus vector type 2 transduction in the liver

Human gene therapy, 2005-03, Vol.16 (3), p.299-306

2005

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31. AAV Gene Therapy for Choroideremia: Dose Determination Analyses

Molecular therapy, 2016-05, Vol.24, p.S14-S14

2016

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Corrigendum to "Inadvertent Germline Transmission of AAV2 Vector: Findings in a Rabbit Model Correlate with Those in a Human Clinical Trial"

Molecular therapy, 2006-12, Vol.14 (6), p.893-893

2006

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1034. Expression of Short Hairpin RNAs by Liver and Non Liver Specific RNA Pol II Expression Cassettes: What Governs Activity?

Molecular therapy, 2006-05, Vol.13 (S1), p.S397-S397

2006

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Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

Viral vector-mediated RNA interference

Preclinical Evaluation of An Anti-HCV miRNA Cluster for Treatment of HCV Infection

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Posttranslational modifications of recombinant myotube-synthesized human factor IX

Inhibition of hepatitis C virus replication using adeno‐associated virus vector delivery of an exogenous anti–hepatitis C virus microrna cluster

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

Inadvertent Germline Transmission of AAV2 Vector: Findings in a Rabbit Model Correlate with Those in a Human Clinical Trial

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Preclinical Gene Therapy Studies for Hemophilia Using Adeno-Associated Virus (AAV) Vectors

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

Modified infusion procedures affect recombinant adeno- associated virus vector type 2 transduction in the liver

31. AAV Gene Therapy for Choroideremia: Dose Determination Analyses

Corrigendum to "Inadvertent Germline Transmission of AAV2 Vector: Findings in a Rabbit Model Correlate with Those in a Human Clinical Trial"

1034. Expression of Short Hairpin RNAs by Liver and Non Liver Specific RNA Pol II Expression Cassettes: What Governs Activity?

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