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826 results for “gene therapy”
Dataset: Taysha Gene Therapies, Inc. (TSHA) Stock Performance
This dataset provides historical stock market performance data for specific companies. It enables users to analyze and understand the past trends and fluctuations in stock prices over time. This information can be utilized for various purposes such as investment analysis, financial research, and market trend forecasting.
Bidirectional Regulation of Motor Circuits Using Magnetogenetic Gene Therapy
<p><span>Here we report a novel suite of magnetogenetic tools, based on a single anti-ferritin nanobody-TRPV1 receptor fusion protein, which regulated neuronal activity when exposed to magnetic fields. AAV-mediated delivery of a floxed nanobody-TRPV1 into the striatum of adenosine 2a receptor-cre driver mice resulted in motor freezing when placed in an MRI or adjacent to a transcranial magnetic stimulation (TMS) device. Functional imaging and fiber photometry both confirmed activation of the target region in response to the magnetic fields. Expression of the same construct in the striatum of wild-type mice along with a second injection of an AAVretro expressing cre into the globus pallidus led to similar circuit specificity and motor responses. Finally, a mutation was generated to gate chloride and inhibit neuronal activity. Expression of this variant in subthalamic nucleus in PitX2-cre parkinsonian mice resulted in reduced local c-fos expression and motor rotational behavior. These data demonstrate that magnetogenetic constructs can bidirectionally regulate activity of specific neuronal circuits non-invasively <em>in-vivo</em> using clinically available devices.</span></p>
GDNF gene therapy for alcohol use disorder in male non-human primates
<p>These the minimal data sets that were utilized in analysis and interpretation for the manuscript "GDNF gene therapy treatment for alcohol use disorder" by Matthew Ford et al. These data sets were collected during a study to evaluate the utility of viral-based GDNF expression in the ventral tegmental area to reduce alcohol intake and prevent relapse in a primate model of alcohol use disorder, and ultimately the feasibility of GDNF as a gene therapy for alcohol use disorder. There are data sets on behavior, biochemical analyses, and cyclic voltammetry.</p>
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
ClinicalTrials.gov study NCT03852498. IPD Sharing: YES. Countries: 6. Publications: 1.
A Study of Rucaparib Versus Physician's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency
ClinicalTrials.gov study NCT02975934. IPD Sharing: YES. Countries: 12. Publications: 3.
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
ClinicalTrials.gov study NCT03306277. IPD Sharing: YES. Countries: 1. Publications: 2.
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
ClinicalTrials.gov study NCT03837184. IPD Sharing: YES. Countries: 3. Publications: 1.
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
ClinicalTrials.gov study NCT03461289. IPD Sharing: YES. Countries: 4. Publications: 2.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy
<p>Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vectors (LVs) has emerged as a promising therapeutic option for CGD patients. We performed non-clinical Good Laboratory Practice (GLP) and laboratory-grade studies to assess the safety and genotoxicity of LV targeting myeloid specific Gp91phox expression in X-linked chronic granulomatous disease (XCGD) mice. We found persistence of gene-corrected cells for up to 1 year, restoration of Gp91phox expression and NADPH oxidase activity in XCGD phagocytes, and reduced tissue inflammation after LV-mediated HSPC GT.<br> Although most of the mice showed no hematological or biochemical toxicity, a small subset of XCGD GT mice developed<br> T cell lymphoblastic lymphoma (2.94%) and myeloid leukemia (5.88%). No hematological malignancies were identified in C57BL/6 mice transplanted with transduced XCGD HSPCs. Integration pattern analysis revealed an oligoclonal composition with rare dominant clones harboring vector insertions near oncogenes in mice with tumors. Collectively, our data support the long-term efficacy of LV-mediated HSPC GT in XCGD mice and provide a safety warning because the chronic inflammatory XCGD background may contribute to oncogenesis.</p>
Late gene therapy limits the restoration of retinal function in a mouse model of retinitis pigmentosa
<p><span>Retinitis pigmentosa is an inherited photoreceptor degeneration that begins with rod loss followed by cone loss. This cell loss greatly diminishes vision, with most patients becoming legally blind. Gene therapies are being developed, but it is unknown how retinal function depends on the time of intervention. To uncover this dependence, we utilize a mouse model of retinitis pigmentosa capable of artificial genetic rescue. This model enables a benchmark of best-case gene therapy by removing variables that complicate the ability to answer this vital question. Complete genetic rescue was performed at 25%, 50%, and 70% rod loss (early, mid, and late, respectively). Here we show early- and mid-treatment restores retinal function to near wild-type levels, specifically the sensitivity and signal fidelity of retinal ganglion cells, the output neurons of the retina. However, some anatomical defects persist. Late treatment retinas exhibit continued, albeit slowed, loss of sensitivity and signal fidelity among retinal ganglion cells, as well as persistent gliosis. We conclude that gene replacement therapies delivered after 50% rod loss are unlikely to restore visual function to normal. This is critical information for administering gene therapies to rescue vision.</span></p>
Choroideremia Gene Therapy Clinical Trial
ClinicalTrials.gov study NCT02553135. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Phase 1 Trial of Interleukin 12 Gene Therapy for Locally Recurrent Prostate Cancer
ClinicalTrials.gov study NCT02555397. IPD Sharing: Not stated. Countries: 1. Publications: 2.
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.
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
ClinicalTrials.gov study NCT03507686. IPD Sharing: NO. Countries: 3. Publications: 2.
Effect of Cyclosporine Therapy on Gene Expression in Patients With Large Granular Lymphocyte Leukemia
ClinicalTrials.gov study NCT00363779. IPD Sharing: Not stated. Countries: 1. Publications: 3.
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
ClinicalTrials.gov study NCT02633943. IPD Sharing: YES. Countries: 8. Publications: 3.
Gene Therapy Using Anti-Her-2 Cells to Treat Metastatic Cancer
ClinicalTrials.gov study NCT00924287. IPD Sharing: Not stated. Countries: 1. Publications: 3.
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
ClinicalTrials.gov study NCT04240314. IPD Sharing: Not stated. Countries: 1. Publications: 5.
Safety Study of Gene Therapy for Ischemic Heart Disease in Korea
ClinicalTrials.gov study NCT01422772. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
ClinicalTrials.gov study NCT03252847. IPD Sharing: NO. Countries: 2. Publications: 1.
ScienceDex guides
Understand access before you commit
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.