An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy

Analysis of AAV-Extracted DNA by Charge Detection Mass Spectrometry Reveals Genome Truncations

(2023) Lauren F. Barnes et al.   Analytical Chemistry

Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity

(2022) Ngoc Tam Tran et al.   HUMAN GENE THERAPY

Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps

(2022) Mark A. Brimble et al.   Molecular Therapy-Methods & Clinical Development

In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency

(2022) Claudia Huichalaf et al.   Molecular Therapy-Methods & Clinical Development

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

(2022) Denise E. Sabatino et al.   MOLECULAR THERAPY

Flies in the ointment: AAV vector preparations and tumor risk

(2021) Junping Zhang et al.   MOLECULAR THERAPY

Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

(2021) Xavier Palazzi et al.   HUMAN GENE THERAPY

Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors

(2021) Julie A. Hutt et al.   TOXICOLOGIC PATHOLOGY

Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV

(2020) Leon Morales et al.   MOLECULAR THERAPY

High-resolution histological landscape of AAV DNA distribution in cellular compartments and tissues following intramuscular and intravenous injection

(2020) Junling Zhao et al.   Molecular Therapy-Methods & Clinical Development

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

(2020) Deepa Chand et al.   JOURNAL OF HEPATOLOGY

Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

(2020) Deepa H. Chand et al.   JOURNAL OF PEDIATRICS

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

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

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

(2019) Natalie F. Nidetz et al.   PHARMACOLOGY & THERAPEUTICS

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Pharmacology of Recombinant Adeno-associated Virus Production

(2018) Magalie Penaud-Budloo et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

(2018) Phillip W.L. Tai et al.   Molecular Therapy-Methods & Clinical Development

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

(2018) Basel T. Assaf et al.   TOXICOLOGIC PATHOLOGY

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

(2017) Hildegund C.J. Ertl et al.   HUMAN GENE THERAPY

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

(2017) Diane Golebiowski et al.   HUMAN GENE THERAPY

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/− Rhesus Macaques

(2017) Jenny A. Greig et al.   Human Gene Therapy Clinical Development

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

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

(2012) David A Suhy et al.   MOLECULAR THERAPY

Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

(2011) Lili Wang et al.   HUMAN GENE THERAPY

Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates

(2011) Hua Li et al.   MOLECULAR THERAPY

AAV8-mediated Hepatic Gene Transfer in Infant Rhesus Monkeys (Macaca mulatta)

(2011) Lili Wang et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses

(2009) Guangping Gao et al.   HUMAN GENE THERAPY