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MetabolomicsCaridac untargeted lipidomics in Sham or TAC-operated mice pretreated with AAV9-NC or AAV9-cTnT-shSlc9a3

Background: Heart failure with reduced ejection fraction (HFrEF) is characterized by impaired contractility and high mortality. Dysregulation of intracellular ion cycling underlies the decline in cardiac contractility. Modulation of cardiac Na+/H+ and Ca2+ handling is consider...

2026-02-20 | MTBLS13701 | MetaboLights
TranscriptomicsGenotoxicity after adeno-associated virus (AAV) gene therapy is dependent upon dose, treatment age and enhancer-promoter selection
AAV gene therapy has recently been approved for clinical use and shown to be efficacious and safe in a growing number of clinical trials. However, the safety of AAV as a gene therapy has been challenged by a few studies that documented hepatocellular carcinoma (HCC) after AAV gene delivery in mice. ...
ORGANISM(S): Mus musculus 
2015-01-01 | E-GEOD-57597 | biostudies-arrayexpress
ProteomicsProteome Analysis of AAV producing HEK293 cells
Adeno-associated viral (AAV) vectors are widely used for gene therapy, providing treatment for diseases caused by absent or defective genes. Despite the success of gene therapy, AAV-manufacturing is still challenging, with production yields being limited. With increased patient demand, improvements ...
ORGANISM(S): Homo sapiens (Human) 
2021-12-09 | PXD028154 | Pride
Hek293 Proteomics Gene therapy Aav Lc-ms/ms Label free 
TranscriptomicsGenotoxicity after adeno-associated virus (AAV) gene therapy is dependent upon dose, treatment age and enhancer-promoter selection (microRNA)
AAV gene therapy has recently been approved for clinical use and shown to be efficacious and safe in a growing number of clinical trials. However, the safety of AAV as a gene therapy has been challenged by a few studies that documented hepatocellular carcinoma (HCC) after AAV gene delivery in mice. ...
ORGANISM(S): Mus musculus 
2015-09-01 | E-GEOD-61632 | biostudies-arrayexpress
TranscriptomicsA multi-functional AAV-CRISPR-Cas9 and its host response
CRISPR-Cas9 delivery by AAV holds promise for gene therapy but faces critical barriers due to its potential immunogenicity and limited payload capacity. Here, we demonstrate genome engineering in postnatal mice using AAV-split-Cas9, a multi-functional platform customizable for genome-editing, transc...
ORGANISM(S): Mus musculus 
2016-07-29 | E-GEOD-84534 | biostudies-arrayexpress
TranscriptomicsResponse of mouse liver to Adenovirus, Helper Dependent Adenovirus, and Adeno-Associated Virus
Understanding host responses to viral gene therapy vectors is necessary for the development of safe and efficacious in vivo gene transfer agents. We describe the use of high-density spotted complementary DNA microarrays in monitoring the in vivo host transcriptional responses in mouse liver upon adm...
ORGANISM(S): Mus musculus 
2010-06-25 | E-GEOD-16047 | biostudies-arrayexpress
ProteomicsFast & High Throughput LC-MS Characterization, and Peptide Mapping of Engineered AAV Capsids Using LC-MS/MS
Adeno-associated virus (AAV) has emerged as a leading platform for gene therapy. With a skyrocketing rate of AAV research and the prevalence of many new engineered capsids being investigated in preclinical and clinical trials, capsid characterization plays an important role in serotype confirmation ...
ORGANISM(S): Adeno-associated virus 9 
2023-03-11 | PXD035837 | Pride
Peptide map. Aav capsids Engineered aav capsids Lcms Msms 
ProteomicsSplit intein mdx skeletal muscle proteomics
Duchenne muscular dystrophy is a rare and lethal neuromuscular disease caused by loss-of-function mutations in the dystrophin protein that provides structural integrity to striated muscle fibers. Mice with loss-of-function mutations for the Dmd gene encoding dystrophin (mdx-4cv) were treated with mi...
ORGANISM(S): Mus musculus (Mouse) 
2025-11-06 | PXD062324 | Pride
Aav therapy Split-intein Duchenne muscular dystrophy Skeletal muscle Lcms 
TranscriptomicsmiRNA and mRNA expression profiling of hepatocellular carcinoma induced by AAV in vivo gene targeting at the Rian locus
This SuperSeries is composed of the following subset Series: GSE35402: miRNA expression profiling of hepatocellular carcinoma induced by AAV in vivo gene targeting at the Rian locus GSE35403: mRNA expression profiling of hepatocellular carcinoma induced by AAV in vivo gene targeting at the Rian locu...
ORGANISM(S): Mus musculus 
2012-07-02 | E-GEOD-35404 | biostudies-arrayexpress
GenomicsCommon AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue
Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue
PRJNA1241144 | ENA
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