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826
datasets available to search
ShareScore release 0.9.0
Dataset results
826 results for “gene therapy”
Randomized Trial of Suicide Gene Therapy and Prostate Cancer
ClinicalTrials.gov study NCT00583492. IPD Sharing: Not stated. Countries: 1. Publications: 5.
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
ClinicalTrials.gov study NCT03861273. IPD Sharing: YES. Countries: 16. Publications: 2.
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
ClinicalTrials.gov study NCT04628585. IPD Sharing: YES. Countries: 2. Publications: 1.
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
ClinicalTrials.gov study NCT02168686. IPD Sharing: Not stated. Countries: 1. Publications: 1.
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
ClinicalTrials.gov study NCT03061201. IPD Sharing: YES. Countries: 1. Publications: 2.
Safety and Efficacy Study Using Gene Therapy for Critical Limb Ischemia
ClinicalTrials.gov study NCT01064440. IPD Sharing: Not stated. Countries: 2. Publications: 1.
Gene Therapy for Achromatopsia (CNGB3)
ClinicalTrials.gov study NCT03001310. IPD Sharing: NO. Countries: 2. Publications: 1.
CYP19A1 (Cytochrome P450 Family 19 Subfamily A Member 1) Gene and Pharmacogenetics of Response to Testosterone Therapy
ClinicalTrials.gov study NCT01378299. IPD Sharing: YES. 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.
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
ClinicalTrials.gov study NCT00430625. IPD Sharing: Not stated. Countries: 5. Publications: 1.
Safety and Efficacy of Injectable Klotho Plasmid Gene Therapy in Humans
ClinicalTrials.gov study NCT07216781. IPD Sharing: YES. Countries: 2. Publications: 14.
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov study NCT05096221. IPD Sharing: Not stated. Countries: 9. Publications: 5.
Late gene therapy limits the restoration of retinal function in a mouse model of retinitis pigmentosa
Open the record for dataset details and reuse information.
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>
Integrome signatures of lentiviral gene therapy for SCID-X1 patients
<p>Files:<br> Supplemental_Table1.xlsx. Recurrent integration genes in all SCID-X1 patients<br> Supplemental_Table2.xlsx. List of all ENCODE datasets used in the analysis<br> Supplementary_Data1_zip: Vector integration sites and their abundance in SCID-X1 patients and CAR T–cell samples</p>
THOR - Tübingen Choroideremia Gene Therapy Trial
ClinicalTrials.gov study NCT02671539. IPD Sharing: NO. Countries: 1. Publications: 1.
Assessing the Suitability of an Imaging Probe for Use in Clinical Cell and Gene Therapy Trials in Cancer and Rheumatoid Arthritis
ClinicalTrials.gov study NCT00185848. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Phase II High Risk Prostate Cancer Trial Using Gene & Androgen Deprivation Therapies, Radiotherapy, & Surgery
ClinicalTrials.gov study NCT03541928. IPD Sharing: UNDECIDED. Countries: 1. Publications: 12.
Gene Therapy for Blindness Caused by Choroideremia
ClinicalTrials.gov study NCT01461213. IPD Sharing: Not stated. Countries: 1. Publications: 4.
Desiccated Thyroid Extract Versus Synthetic T3/T4 Combination (ThyrolarTM) Versus L-T4 Alone in the Therapy of Primary Hypothyroidism With Special Attention to the Gene Polymorphism
ClinicalTrials.gov study NCT02317926. IPD Sharing: Not stated. Countries: 1. Publications: 1.
ScienceDex guides
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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.