Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression

Safety, efficacy and biodistribution of an engineered hepatotropic Adeno-Associated Virus mediated gene therapy for Ornithine Transcarbamylase Deficiency in Cynomolgus monkeys

(2021) Julien Baruteau et al.   Molecular Therapy-Methods & Clinical Development

Immune Responses to Viral Gene Therapy Vectors

(2020) Jamie L. Shirley et al.   MOLECULAR THERAPY

Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?

(2020) J. Fraser Wright   MOLECULAR THERAPY

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2020) Giuseppe Ronzitti et al.   Frontiers in Immunology

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression

(2020) Barbara A Konkle et al.   BLOOD

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9

(2019) Zheng Chai et al.   HUMAN GENE THERAPY

Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells

(2019) Jamie L. Shirley et al.   MOLECULAR THERAPY

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

(2018) Lili Wang et al.   NATURE BIOTECHNOLOGY

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors

(2018) Roberto Calcedo et al.   Human Gene Therapy Methods

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

(2018) Klaudia Kuranda et al.   JOURNAL OF CLINICAL INVESTIGATION

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

(2018) Jenny A. Greig et al.   Molecular Therapy-Methods & Clinical Development

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Long-Term Sex-Biased Correction of Circulating Propionic Acidemia Disease Markers by Adeno-Associated Virus Vectors

(2015) Adam J. Guenzel et al.   HUMAN GENE THERAPY

Genotype–Phenotype Correlations in Ornithine Transcarbamylase Deficiency: A Mutation Update

(2015) Ljubica Caldovic et al.   Journal of Genetics and Genomics

Long-term outcomes in Ornithine Transcarbamylase deficiency: a series of 90 patients

(2015) Anais Brassier et al.   Orphanet Journal of Rare Diseases

Critical Role of Interferon-α Constitutively Produced in Human Hepatocytes in Response to RNA Virus Infection

(2014) Yoji Tsugawa et al.   PLoS One

Occurrence of Multinucleated Hepatocytes in Cynomolgus Monkeys (Macaca fascicularis) From Different Geographical Regions

(2014) M. N. Novilla et al.   VETERINARY PATHOLOGY

The incidence of urea cycle disorders

(2013) Marshall L. Summar et al.   MOLECULAR GENETICS AND METABOLISM

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

(2011) C. Binny et al.   BLOOD

Hyperammonemic coma in an ornithine transcarbamylase mutation carrier following antepartum corticosteroids

(2011) S Lipskind et al.   Journal of Perinatology

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

(2011) Lili Wang et al.   MOLECULAR GENETICS AND METABOLISM

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation

(2009) Allison P Dane et al.   MOLECULAR THERAPY

Enhancing transduction of the liver by adeno-associated viral vectors

(2008) A C Nathwani et al.   GENE THERAPY