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Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

Human gene therapy. Part B. Methods, 2012-02, Vol.23 (1), p.1-7

2012

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The intersection of vector biology, gene therapy, and hemophilia

Research and practice in thrombosis and haemostasis, 2021-08, Vol.5 (6), p.e12586-n/a, Article e12586

2021

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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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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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AAV vector production: Troublesome host innate responses in another setting

Molecular therapy. Methods & clinical development, 2023-09, Vol.30, p.332-332

Wright, J. Fraser

2023

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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Ophthalmology (Rochester, Minn.), 2019-09, Vol.126 (9), p.1273-1285

2019

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Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography

Journal of virological methods, 2007-03, Vol.140 (1), p.183-192

2007

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The promising potential of gene therapy for diabetes mellitus

Nature reviews. Endocrinology, 2024-11, Vol.20 (11), p.627-628

2024

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Safe, Long-term Hepatic Expression of Anti-HCV shRNA in a Nonhuman Primate Model

Molecular therapy, 2012-09, Vol.20 (9), p.1737-1749

2012

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Manufacturing and regulatory strategies for clinical AAV2-hRPE65

Current gene therapy, 2010-10, Vol.10 (5), p.341-349

2010

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Progress and challenges in viral vector manufacturing

Human molecular genetics, 2016-04, Vol.25 (R1), p.R42-R52

2016

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Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

Molecular therapy, 2009-01, Vol.17 (1), p.144-152

2009

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Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

Molecular therapy, 2010-07, Vol.18 (7), p.1318-1329

2010

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Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model

Human gene therapy, 2011-07, Vol.22 (7), p.843-852

2011

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Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors

Molecular therapy, 2009-06, Vol.17 (6), p.1022-1030

2009

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Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

Molecular therapy. Methods & clinical development, 2014-01, Vol.1 (C), p.14036-14036, Article 14036

2014

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In Vitro Characterization of the Activity of PF-05095808, a Novel Biological Agent for Hepatitis C Virus Therapy

Antimicrobial Agents and Chemotherapy, 2012-03, Vol.56 (3), p.1364-1375

2012

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PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9

Journal of virological methods, 2011-04, Vol.173 (1), p.99-107

2011

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Design of experiments as a decision tool for cell therapy manufacturing

Cytotherapy (Oxford, England), 2022-06, Vol.24 (6), p.590-596

2022

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AAV2 gene therapy readministration in three adults with congenital blindness

Science translational medicine, 2012-02, Vol.4 (120), p.120ra15

2012

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Recombinant Adeno-Associated Virus Quality Control for Non-Clinical and Clinical Vectors: How an Unregulated Commercial Sector Can Compromise Development of New Gene Therapies

Human gene therapy, 2019-12, Vol.30 (12), p.1447-1448

2019

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Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.15056-15056, Article 15056

2016

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Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Molecular therapy, 2010-03, Vol.18 (3), p.643-650

2010

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Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial

The lancet HIV, 2019-04, Vol.6 (4), p.e230-e239

2019

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Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

The intersection of vector biology, gene therapy, and hemophilia

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

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

AAV vector production: Troublesome host innate responses in another setting

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography

The promising potential of gene therapy for diabetes mellitus

Safe, Long-term Hepatic Expression of Anti-HCV shRNA in a Nonhuman Primate Model

Manufacturing and regulatory strategies for clinical AAV2-hRPE65

Progress and challenges in viral vector manufacturing

Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model

Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

In Vitro Characterization of the Activity of PF-05095808, a Novel Biological Agent for Hepatitis C Virus Therapy

PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9

Design of experiments as a decision tool for cell therapy manufacturing

AAV2 gene therapy readministration in three adults with congenital blindness

Recombinant Adeno-Associated Virus Quality Control for Non-Clinical and Clinical Vectors: How an Unregulated Commercial Sector Can Compromise Development of New Gene Therapies

Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial

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