Contact and support
Need help, have a question, or want to contact the ResearchHub team?
© 2026 ResearchHub. Built for responsible scholarly connection.
Privacy-aware community infrastructureResearch graph
References from DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery. Local targets link to admitted publications; unresolved targets remain external evidence.
Adeno-associated virus vector as a platform for gene therapy delivery
10.1038/s41573-019-0012-9 · 2019 · External reference
The state of cell and gene therapy in 2023
10.1016/j.ymthe.2023.11.001 · 2023 · External reference
The progress of AAV-mediated gene therapy in neuromuscular disorders
10.1080/14712598.2018.1479739 · 2018 · External reference
Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape
10.3390/ijms241813743 · 2023 · External reference
Spinal Muscular Atrophy: Past, Present, and Future
10.1542/neo.20-8-e437 · 2019 · External reference
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10.1056/nejmoa1706198 · 2017 · External reference
Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
10.1016/j.jhep.2020.11.001 · 2021 · External reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
Addressing high dose AAV toxicity - “one and done” or “slower and lower”
10.1080/14712598.2022.2060737 · 2022 · External reference
Real-world safety data of the orphan drug onasemnogene abeparvovec (Zolgensma®) for the SMA rare disease: a pharmacovigilance study based on the EMA adverse event reporting system
10.3390/ph17030394 · 2024 · External reference
Comprehensive analysis of adverse events associated with onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy patients: insights from FAERS database
2024 · External reference
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
10.1089/hum.2018.015 · 2018 · External reference
Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles
10.1089/hum.2021.116 · 2022 · External reference
Liver injury in cynomolgus monkeys following intravenous and intrathecal scAAV9 gene therapy delivery
10.1016/j.ymthe.2023.07.020 · 2023 · External reference
AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain
10.1038/s41467-025-58778-3 · 2025 · External reference
Mechanisms, regulation and functions of the unfolded protein response
10.1038/s41580-020-0250-z · 2020 · External reference
Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain
10.1016/j.neulet.2017.11.049 · 2018 · External reference
Endoplasmic reticulum stress actively suppresses hepatic molecular identity in damaged liver
10.15252/msb.20199156 · 2020 · External reference
Gene Expression Predicts Histological Severity and Reveals Distinct Molecular Profiles of Nonalcoholic Fatty Liver Disease
10.1038/s41598-019-48746-5 · 2019 · External reference
Hepatocyte Nuclear Factor 4 Alpha: A Key Regulator of Liver Disease Pathology and Haemostatic Disorders
10.1111/liv.16245 · 2025 · External reference
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury
10.1016/j.ymthe.2024.02.002 · 2024 · External reference
Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy
10.1016/j.ymthe.2025.05.017 · 2025 · External reference
Death after high-dose rAAV9 gene therapy in a patient with Duchenne’s muscular dystrophy
10.1056/nejmoa2307798 · 2023 · External reference
Tauroursodeoxycholic acid reduces ER stress by regulating of Akt-dependent cellular prion protein
10.1038/srep39838 · 2016 · External reference
Trimmomatic: a flexible trimmer for Illumina sequence data
10.1093/bioinformatics/btu170 · 2014 · External reference
STAR: ultrafast universal RNA-seq aligner
10.1093/bioinformatics/bts635 · 2013 · External reference
featureCounts: an efficient general purpose program for assigning sequence reads to genomic features
10.1093/bioinformatics/btt656 · 2014 · External reference
edgeR: a Bioconductor package for differential expression analysis of digital gene expression data
10.1093/bioinformatics/btp616 · 2010 · External reference
limma powers differential expression analyses for RNA-sequencing and microarray studies
10.1093/nar/gkv007 · 2015 · External reference
clusterProfiler 4.0: A universal enrichment tool for interpreting omics data
2021 · External reference
Gene set enrichment analysis: A knowledge-based approach for interpreting genome-wide expression profiles
10.1073/pnas.0506580102 · 2005 · External reference
