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212 results for “AAV”
Systematic Multi-Trait AAV Capsid Engineering for Efficient Gene Delivery
<p>Datasets for "Systematic Multi-Trait AAV Capsid Engineering for Efficient Gene Delivery", Eid et al., <em>Nature Communications. </em></p>
Ca2+ activity maps of astrocytes tagged by axo-astrocytic AAV transfer
<p>Astrocytes exhibit localized Ca<sup>2+</sup> microdomain (MD) activity thought to be actively involved in information processing in the brain. However, functional organization of Ca<sup>2+</sup> MDs in space and time in relationship to behavior and neuronal activity is poorly understood. Here, we first show that Adeno-Associated Virus (AAV) particles transfer anterogradely from axons to astrocytes. Then we use this axo-astrocytic AAV transfer to express genetically encoded Ca<sup>2+</sup> indicators at high contrast circuit-specifically. In combination with two-photon microscopy and unbiased, event-based analysis we investigated cortical astrocytes embedded in the vibrissal thalamocortical circuit. We found a wide range of Ca<sup>2+</sup> MD signals, some of which were ultrafast (≤300 ms). Frequency and size of signals were extensively increased by locomotion but only subtly with sensory stimulation. The overlay of these signals resulted in behavior dependent maps with characteristic Ca<sup>2+</sup> activity hotspots, maybe representing memory engrams. These functional subdomains are stable over days, suggesting subcellular specialization.</p>
Data for: An AAV-CRISPR/Cas9-strategy for gene editing across divergent rodent species: Targeting neural oxytocin receptors as a proof of concept
<p>A major issue in neuroscience is the poor translatability of research results from preclinical studies in animals to clinical outcomes. Comparative neuroscience can overcome this barrier by studying multiple species to differentiate between species-specific and general mechanisms of neural circuit functioning. Targeted manipulation of neural circuits often depends on genetic dissection, and use of this technique has been restricted to only a few model species, limiting its application in comparative research. However, ongoing advances in genomics make genetic dissection attainable in a growing number of species. To demonstrate the potential of comparative gene editing approaches, we developed a viral-mediated CRISPR/Cas9 strategy that is predicted to target the oxytocin receptor (<em>Oxtr</em>) gene in >80 rodent species. This strategy specifically reduced OXTR levels in all evaluated species (n=6), without causing gross neuronal toxicity. Thus, we show that CRISPR/Cas9-based tools can function in multiple species simultaneously. Thereby, we hope to encourage comparative gene editing and improve the translatability of neuroscientific research.</p>
Raw FITS and AAV data of the Bluewalker 3 LEO communication satellite.
<p>Separate tar/zip files of raw images from each telescope used in the l2022 observing campaign of the AST SpaceMobile prototype Bluewalker 3 LEO communication satellite, led and organised by the International Astronomical Union (IAU) <a href="https://cps.iau.org/">Centre for the Protection of the Dark and Quiet Sky from Satellite Constellation Interference</a> (CPS). The images along with their associated calibration frames were used in the publication (Nandakumar+ <a href="https://doi.org/10.21203/rs.3.rs-2557594/v1">2023</a>) and are freely available to the community. The reduced data (e.g. apparent magnitude, TLE accuracy, and phase angles) are provided in (Nandakumar+ <a href="https://doi.org/10.21203/rs.3.rs-2557594/v1">2023</a>) as supplementary machine readable data tables.</p>
Targeting AAV vectors to the CNS via de novo engineered capsid-receptor interactions
<p>Dataset for "Targeting AAV vectors to the CNS via <em>de novo</em> engineered capsid-receptor interactions."</p>
Ca2+ activity maps of astrocytes tagged by axo-astrocytic AAV transfer
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Data from: Double-strand break repair pathways differentially affect processing and transduction by dual AAV vectors
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Data for: An AAV-CRISPR/Cas9-strategy for gene editing across divergent rodent species: Targeting neural oxytocin receptors as a proof of concept
Open the record for dataset details and reuse information.
Data from: Significant differences in capsid properties and potency between AAV vectors produced in Sf9 and HEK293 cells
<p>For successful vector-based gene therapy manufacturing, the selected adeno-associated virus (AAV) vector production system must produce vector at sufficient scale. However, concerns have arisen regarding the quality of vector produced using different systems. In this study, we compared AAV serotypes 1, 8, and 9 produced by two different systems (Sf9/baculovirus and HEK293/transfection) and purified by two separate processes. We evaluated capsid properties including protein composition, post-translational modification, particle content profiles, and in vitro and in vivo vector potency. Vectors produced in the Sf9/baculovirus system displayed reduced incorporation of viral protein 1 and 2 into the capsid, increased capsid protein deamidation, increased empty and partially packaged particles in vector preparations, and an overall reduced potency. The differences observed were largely independent of the harvest method and purification process. These findings illustrate the need for careful consideration when choosing an AAV vector production system for clinical production.</p>
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
ClinicalTrials.gov study NCT00749957. IPD Sharing: Not stated. Countries: 1. Publications: 5.
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
ClinicalTrials.gov study NCT04669535. IPD Sharing: NO. Countries: 1. Publications: 2.
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.
Data from: CRISPR screening by AAV episome-sequencing (CrAAVe-seq): A scalable cell type-specific in vivo platform uncovers neuronal essential genes
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Data from: Significant differences in capsid properties and potency between AAV vectors produced in Sf9 and HEK293 cells
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AAV-mediated Inner ear gene delivery triggers mild host immune responses in the mammalian inner ear
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Data from: Development of an adipose-tropic AAV capsid ablating liver tropism
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Mechanisms of AAV neutralization by human alpha-defensins
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Enhancing gene transfer to renal tubules and podocytes by context-dependent selection of AAV capsids
<p>RawData.zip file provides AAV Barcode-Seq raw read count data used for Figure 1 and Supplementary Figure 1. Each file represents NGS read count data of each barcode (BC) PCR product indicated in the file name. VBC_AAV_capsid_Conversion_Table.csv provides information about the barcodes, CAGBC101 to CAGBC223, used for AAV capsids contained in the library. </p>
AAV gene therapy encoding equine IL10
<p>Equine recurrent uveitis (ERU) is a spontaneous, painful, and vision threatening disease affecting up to 25% of equine populations worldwide. Current treatments of ERU are non-specific and have many side effects which limits them to short-term use. In order to develop an effective therapy for ERU, we investigated the use of adeno-associated virus (AAV) gene therapy, exploiting a natural immune tolerance mechanism induced by equine interleukin-10 (Equine-IL10). The purpose of this study was to evaluate the therapeutic efficacy of a single intravitreal (IVT) dose of AAV8-Equine-IL10 gene therapy for inhibition of experimental autoimmune uveitis (EAU) in rats. Each rat was dosed intravitreally (IVT) in both eyes with either balanced salt solution (BSS) (control; n=4), AAV8-Equine-IL10 at a low dose (2.4x10<sup>9</sup> vg; n=5) or high dose (2.4x10<sup>10</sup> vg; n=5). EAU was induced in all groups of rats 7 days after IVT injections and euthanized 21 days post-injection. Ophthalmic examination and aqueous humor (AH) cell counts were recorded with the observer blinded to the treatment groups. Histopathology and qPCR were performed on selected ocular tissues. Data presented herein demonstrate that AAV8-Equine-IL10 treated rats exhibited a significant decrease in clinical inflammatory scores and AH cell counts compared to BSS-treated EAU eyes on days 10, 12 and 14 post EAU induction at both administered vector doses. Mean cellular histologic infiltrative scores were also significantly less in AAV8-Equine-IL10 dosed rats compared to the BSS group. Intravitreal injection of AAV8-Equine-IL10 resulted in Equine-IL10 cDNA expression in the ciliary body, retina, cornea, and optic nerve in a dose-dependent manner. A single IVT injection of AAV8-Equine-IL10 appeared to be well-tolerated and inhibited EAU even at the lowest administered dose. These results demonstrate safety and efficacy of AAV8-Equine-IL10 to prevent EAU and support continued exploration of AAV gene therapy for the treatment of equine and perhaps human recurrent uveitis.</p>
A Phase 1, Randomized, Blinded, Dose-escalation Study of rAAV1-PG9DP Recombinant AAV Vector Coding for PG9 Antibody in Healthy Male Adults.
ClinicalTrials.gov study NCT01937455. IPD Sharing: Not stated. Countries: 1. Publications: 2.
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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.