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42 results for “Adeno-associated virus”
Adeno-associated virus type 2 in children from the United States with acute severe hepatitis
<p>This repository contains the metadata and code used in the analysis of the manuscript, <em>Adeno-associated virus type 2 in children from the United States with acute severe hepatitis.</em></p>
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
ClinicalTrials.gov study NCT02161380. IPD Sharing: Not stated. Countries: 1. Publications: 11.
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
ClinicalTrials.gov study NCT01687608. IPD Sharing: YES. Countries: 1. Publications: 2.
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
ClinicalTrials.gov study NCT00976352. IPD Sharing: NO. Countries: 1. Publications: 9.
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
ClinicalTrials.gov study NCT05788536. IPD Sharing: YES. Countries: 4. Publications: 1.
Isolating human monoclonal antibodies against adeno-associated virus from donors with pre-existing immunity
<p>With the advent of single B-cell cloning technology, we can isolate antibodies against virtually any antigen to study the interaction of a given pathogen with the immune system and develop novel therapeutic strategies. Antibodies directed against the capsid of adeno-associated viruses (AAV) are a significant obstacle to effectively leveraging AAV as a gene-delivery vector in seropositive individuals. In order to design next-generation vectors that can evade neutralization by these antibodies, studies have mapped the epitopes of mousemonoclonal antibodies generated by immunization with AAV. Although these studies provide critical information regarding capsid immunogenicity, they cannot address (1) differences in the antibody repertoire generated in humans following AAV natural infection; or (2) how reactions can vary when generated in response to vector administration. Here, we isolated and evaluated a panel of novel, fully human anti-AAV antibodies by cloning single memory B cells from a seropositive normal donor. We have validated the utility of this approach to study AAV immunology. Our goal is to leverage this knowledge to design novel AAV variants that can effectively transduce target tissues in individuals with AAV-neutralizing antibodies.</p>
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
ClinicalTrials.gov study NCT05518188. IPD Sharing: YES. Countries: 1. Publications: 1.
Safety and Immunogenicity Study of tgAAC09, an HIV Vaccine in an Adeno-associated Virus (AAV) Capsid
ClinicalTrials.gov study NCT00888446. IPD Sharing: Not stated. Countries: 3. Publications: 1.
Isolating human monoclonal antibodies against adeno-associated virus from donors with pre-existing immunity
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Gene Delivery to Neurons in the Auditory Brainstem of Barn Owls using Standard Recombinant Adeno-associated Virus Vectors
<div> <p>Recombinant adeno-associated virus (rAAV) vectors are a commonly used tool for gene delivery. There is a large choice of different serotypes whose transduction efficiency varies for different animal species. In this study, three rAAV vectors were tested for transduction efficiency in the auditory brainstem of adult barn owls (<i>Tyto alba</i>) which are not standard laboratory animals. Injections with rAAV serotypes 2/1 and 2/5 resulted in reliable expression in various nuclei of the auditory brainstem of barn owls. Both vectors showed evidence of being spread by axonal transport. However, only rAAV2/5 also showed expression in regions far distant from the injection site, suggesting long-range axonal transport in connections along the auditory pathway. In contrast, injections with rAAV2/9 resulted in no expression. Our results demonstrate for the first time that commercially available rAAV vectors can be used for reliable gene expression in the barn owl auditory brainstem and pave the way toward optogenetic manipulation of neural activity in this important animal species in neuroethology and auditory physiology.</p> </div>
Dataset 1:Coarse-grained simulations of adeno-associated virus and its receptor reveal influences on membrane lipid organisation and curvature.
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Dataset 2:Coarse-grained simulations of adeno-associated virus and its receptor reveal influences on membrane lipid organisation and curvature.
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Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
ClinicalTrials.gov study NCT06003387. IPD Sharing: YES. Countries: 12. Publications: 0.
Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
ClinicalTrials.gov study NCT00004533. IPD Sharing: Not stated. Countries: 0. Publications: 1.
Gene Delivery to Neurons in the Auditory Brainstem of Barn Owls using Standard Recombinant Adeno-associated Virus Vectors
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The Adeno-Associated Virus 2 (AAV2) genome and Rep 68/78 proteins interact with cellular sites of DNA damage.
GEO Series GSE178316. Homo sapiens. 48 samples. Type: Genome binding/occupancy profiling by high throughput sequencing.
Adeno-associated virus-mediated delivery of anti-miR-199a tough decoys attenuates cardiac hypertrophy
GEO Series GSE155545. Mus musculus. 6 samples. Type: Expression profiling by high throughput sequencing.
Adeno-associated virus-mediated trastuzumab delivery to the central nervous system for human epidermal growth factor receptor 2+ brain metastasis
GEO Series GSE255027. Macaca mulatta. 4 samples. Type: Expression profiling by high throughput sequencing.
CRISPR-SaCas9-KKH targeting of miR-21 through a single adeno-associated virus vector prolongs survival of GB-bearing mice
GEO Series GSE277282. Mus musculus. 57 samples. Type: Other.
A novel human heart-derived natural adeno-associated virus capsid combines cardiospecificity with cardiotropism in vivo
GEO Series GSE290777. Mus musculus. 1 samples. Type: Expression profiling by high throughput sequencing.
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These curated guides explain access requirements, typical timelines, costs, and reuse considerations for widely used research datasets.
Allen Brain Atlas
Allen Brain Atlas is an Allen Institute collection of brain map atlases, datasets, APIs, and analysis tools covering mouse, human, and non-human primate brain resources.
Annotated Behaviour and Observability Dataset (ABODe)
ABODe is a University of Edinburgh DataShare dataset for behavior classification in group-housed mice using home-cage video, identities, bounding boxes, ground-plate positions, and annotator labels.
DANDI Archive for NWB datasets
DANDI is a BRAIN Initiative archive for publishing and sharing neurophysiology data, including electrophysiology, optophysiology, and behavioral data packaged as NWB and related standards.
International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.