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5,016 results for “THERAPEUTICS”
The c-Abl inhibitor IkT-148009 therapeutically suppresses neurodegeneration in models of heritable and sporadic Parkinson's Disease
<p>Parkinson's Disease (PD) is the second most prevalent neurodegenerative disease of the central nervous system, with an estimated 5,000,000 cases worldwide. PD pathology is characterized by the accumulation of misfolded a-synuclein, which is thought to play a critical role in the etiopathogenesis of the disease. Animal models of PD suggest that activation of the Abelson Tyrosine Kinase, or c-Abl, plays an essential role in the initiation and progression of a-synuclein pathology and initiates processes leading to the degeneration of dopaminergic and non-dopaminergic neurons. Given the essential role of c-Abl in the disease, a proprietary c-Abl inhibitor library was developed to identify potent, orally bioavailable c-Abl inhibitors capable of crossing the blood-brain barrier based on pre-defined characteristics, leading to the discovery of IkT-148009. IkT-148009 is a selective, potent, brain-penetrant c-Abl inhibitor with a favorable toxicology profile that was analyzed for therapeutic potential in animal models of slowly progressive, a-synuclein-dependent disease. In models of both inherited and sporadic Parkinson's disease in the mouse, IkT-148009 suppressed c-Abl activation to baseline and substantially protected neurons from degeneration when administered therapeutically by once daily oral gavage beginning four weeks after disease initiation. Recovery of normal behavioral function in diseased mice occurred within 8 weeks of initiating treatment and occurred concomitantly with a substantial reduction of a-synuclein pathology in the brain. These disease-modifying outcomes in mice suggest IkT-148009 has the potential to be a disease-modifying therapy in human disease.</p>
KidDO progress presentation 2022: Metabolic Targets for Therapeutic Intervention in Kidney Disease
<p>This is a recorded talk with head of the KidDO project - Robert Fenton - where he presents the latest project progress.</p> <p>The talk was given at one of ODIN's (the Open Discovery Innovation Network) Knowledge Sharing Events in May 2022.</p> <p> </p> <p> </p>
Imbalanced expression of cation-chloride cotransporters as a potential therapeutic target in an Angelman Syndrome mouse model
<p>We provide 6 files; 1. Data for western blot analysis (WB_NKCC1_KCC2_Fig1A.xlsx), 2. Data for [Cl-]i(Intracelluar‗Cl_Fig1B.xlsx), 3. Electrophysilogical data for mIPSC and tonic current (mIPSC_Tonic current_Fig2B_C_E.xlsx), 4. Data for behavior analysis (Behavior_analysis_Fig3A_C .xlsx), 5. Data for seizure threshhold (seizure_threshold_Fig4A.xlsx), 6. Data for EEG spike number and band power (EEG_analysis_Fig4B_C.xlsx)</p>
Molecular Dynamics Simulation and Docking Studies Reveals Inhibition of NF-kB signaling as a Promising Therapeutic Drug Target for reduction in Cytokines Storms
<p><span>The complexes of the top identified molecules with NF-kB-kB site, as well as all the designed molecules used in the screening process. </span></p>
Modeling Lewy Body Disease with SNCA Triplication iPSC-Derived Cortical Organoids and Identifying Therapeutic Drugs
<p><span>This repository contains the source code for the single-cell and single-nuclei RNA sequencing data analysis for the study <strong><span>Modeling Lewy Body Disease with SNCA Triplication iPSC-Derived Cortical Organoids and Identifying Therapeutic Drugs</span></strong> by Yunjung Jin et al.<br></span></p>
Development of an imaging toolbox to assess the therapeutic potential and biodistribution of macrophages in a mouse model of multiple organ dysfunction
<p>Data set to accompany manuscript entitled "Development of an imaging toolbox to assess the therapeutic potential and biodistribution of regenerative therapies in a mouse model of multiple organ dysfunction " which can be found on BioRxiv.</p>
Internet-based interdisciplinary therapeutic group for perinatal anxiety and depression - a randomized pilot study during COVID-19
<p><strong>Abstract</strong>:</p> <p><strong>Background</strong>: Recent years have seen growing interest in the impact that stress and depressive/anxiety symptomatology have on mothers, fetal development, the mother-baby relationship and infant development. Depressed mothers spend less time making eye-contact with their babies, and caressing and talking to them, which can cause delays in cognitive and emotional development. Early interventions promote reductions in mothers’ stress and anxiety symptoms along with improvements in their ability to read infant cues appropriately. Group perinatal care and internet-based therapeutic interventions were essential during the Covid period and telemedicine ensures continuity of contact with high-risk populations. The aim of this novel study was to assess the effects of interdisciplinary online therapeutic group in at-risk mothers and babies during the Covid-19 pandemic in a disadvantaged neighborhood in Barcelona (Spain).</p> <p><strong>Method</strong>: An interdisciplinary team offered gestating women and mothers comprehensive care in a protective environment in a Primary Health Care Center. Pregnant women and new mothers were screened to detect depressive-anxiety symptomatology through EPDS/STAI questionnaires. A total of 135 babies were born from March 2020 to June 2021, during the pandemic, and 72 women met high-risk criteria for anxiety or depression. Some 37 pregnant women and mother-baby dyads were treated in the therapeutic group and compared with 27 women randomly selected from the control group, who received treatment as usual. A blinded pediatric follow-up was established when the babies were 6 months old to monitor their development through the Llevant Scale. The course of the mothers’ symptomatology was also assessed.</p> <p><strong>Results</strong>: We obtained a significant decrease in depressive-anxiety symptomatology (EPDS p=0.000; STAI state p=0.015 and STAI trait p=0.001 at 6 months of life) in the therapeutic group compared with controls. Follow-up at 6 months demonstrated significant differences between groups in assessment of the babies’ development (manipulation p=0.003; language p=0.000; sociability p=0.000). The therapeutic group showed no difficulties in any areas of development.</p> <p><strong>Conclusions: </strong>Interdisciplinary online therapeutic group helps to ensure healthy development of the baby and reduction of the mothers’ depressive-anxiety symptomatology. Future research should involve larger samples to explore the impact of online group interventions in both mothers and babies.</p> <p><strong>Trial registration: </strong>The study is registered at Jordi Gol i Gurina Foundation number 21/276-P.</p>
Multidimensional control of therapeutic human cell function with synthetic gene circuits
<p>Synthetic gene circuits that precisely control human cell function could expand the capabilities of gene and cell-based therapies. However, platforms for developing circuits in primary human cells that drive robust functional changes in vivo and have compositions suitable for clinical use are lacking. Here, we develop synthetic transcriptional regulators that are compact and based largely on human-derived proteins (synZiFTRs). As a proof of principle, we engineer gene switches and circuits that allow precise, user-defined control over therapeutically-relevant genes in primary T cells using orthogonal, FDA-approved small molecule inducers. Our circuits can instruct T cells to sequentially activate multiple cellular programs, such as proliferation and anti-tumor activity, to drive synergistic therapeutic responses. This platform should accelerate development and clinical translation of synthetic gene circuits in diverse human cell types and contexts.</p>
Dataset related to: Therapeutic Small Interfering RNA Targeting Complement C3 in a Mouse Model of C3 Glomerulopathy
<p>The files contain all the dataset included in the manuscript divided by figures.</p> <p> </p> <p>Abstract</p> <p>Alternative pathway complement dysregulation with abnormal glomerular C3 deposits and glomerular damage is a key mechanism of pathology in C3 glomerulopathy (C3G). No disease-specific treatments are currently available for C3G. Therapeutics inhibiting complement are emerging as a potential strategy for the treatment of C3G. In this study, we investigated the effects of N-acetylgalactosamine (GalNAc) conjugated small interfering RNA (siRNA) targeting the C3 component of complement that inhibits liver C3 expression in the C3G model of mice with heterozygous deficiency of factor H (Cfh+/- mice). We showed a duration of action for GalNAc-conjugated C3 siRNA in reducing the liver C3 gene expression in Cfh+/- mice that were dosed s.c. once a month for up to 7 mo. C3 siRNA limited fluid-phase alternative pathway activation, reducing circulating C3 fragmentation and activation of factor B. Treatment with GalNAc-conjugated C3 siRNA reduced glomerular C3d deposits in Cfh+/- mice to levels similar to those of wild-type mice. Ultrastructural analysis further revealed the efficacy of the C3 siRNA in slowing the formation of mesangial and subendothelial electron-dense deposits. The present data indicate that RNA interference mediated C3 silencing in the liver may be a relevant therapeutic strategy for treating patients with C3G associated with the haploinsufficiency of complement factor H.</p>
Identification of Potential JNK3 Inhibitors Through Virtual Screening, Molecular Docking And Molecular Dynamics Simulation as Therapeutics for Alzheimer's Disease
<p>Alzheimer's disease (AD) is a complex neurological disorder without effective treatment. One factor in its development is c-Jun N-terminal kinases (JNKs), a type of protein related to brain function. JNK3, found mainly in the brain, contributes to AD by promoting brain abnormalities. Current research aims to create new JNK3 inhibitors for AD treatment using a virtual screening method. A database of compounds was filtered, and five potential compounds were identified with better scores than a reference. These compounds underwent simulations and energy calculations, showing stability and potential as JNK3 inhibitors.</p>
Patient Attitudes and Preferences for Outcomes of Inflammatory Bowel Disease Therapeutics
ClinicalTrials.gov study NCT02316678. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Comparison of Therapeutic Effects of Greater Occipital Nerve Block, Topiramate, and Flunarizine on Episodic Migraine
ClinicalTrials.gov study NCT03712917. IPD Sharing: NO. Countries: 1. Publications: 2.
Comparison Of Rituximab Versus Tositumomab and Iodine I 131 Tositumomab (BEXXAR® Therapeutic Regimen) For Patients With Relapsed Follicular Non-Hodgkins Lymphoma
ClinicalTrials.gov study NCT00268983. IPD Sharing: YES. Countries: 3. Publications: 1.
Effectiveness of DECIDE in Patient-Provider Communication, Therapeutic Alliance & Care Continuation
ClinicalTrials.gov study NCT01947283. IPD Sharing: UNDECIDED. Countries: 1. Publications: 1.
A Pilot Study of Intra-arrest Therapeutic Hypothermia in Patients Suffering Non-Traumatic Out of Hospital Cardiac Arrest
ClinicalTrials.gov study NCT01413399. IPD Sharing: Not stated. Countries: 1. Publications: 1.
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
ClinicalTrials.gov study NCT00943111. IPD Sharing: Not stated. Countries: 12. Publications: 7.
Study to IDEntify Patients With Advanced/Metastatic Non Small Cell Lung Cancer (NSCLC) and ALK and ROS1 Translocation and to Establish Their Therapeutic Management (IDEALK&ROS)
ClinicalTrials.gov study NCT02679170. IPD Sharing: NO. Countries: 1. Publications: 1.
Tableted COVID-19 Therapeutic Vaccine
ClinicalTrials.gov study NCT04380532. IPD Sharing: YES. Countries: 2. Publications: 1.
Developing an Online Therapeutic Intervention for Chronic Pain in Veterans
ClinicalTrials.gov study NCT03655132. IPD Sharing: NO. Countries: 1. Publications: 2.
Decision Aid for Therapeutic Options In Sickle Cell Disease
ClinicalTrials.gov study NCT02326597. IPD Sharing: Not stated. Countries: 1. Publications: 9.
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.