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PubMed Journals Articles About "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" - Page: 20 RSS

11:50 EST 20th November 2019 | BioPortfolio

Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides articles that have been published worldwide.

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Showing "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" PubMed Articles 476–500 of 31,000+

Reports of recovered memories of childhood abuse in therapy in France.

Recovered memories of abuse in therapy are especially controversial if the clients were not aware they were abused before therapy. In the past, such memory recovery has led to legal action, as well as a debate about whether such memories might be repressed, forgotten, or false memories. More than two decades after the height of the controversy, it is unclear to what degree such memories are still recovered today, and to what extent it occurs in France. In our French survey of 1312 participants ( = 33; 5...


Integrating Mendelian randomization and multiple-trait colocalization to uncover cell-specific inflammatory drivers of autoimmune and atopic disease.

Immune mediated diseases (IMDs) arise when tolerance is lost and chronic inflammation is targeted towards healthy tissues. Despite their growing prevalence, therapies to treat IMDs are lacking. Cytokines and their receptors orchestrate inflammatory responses by regulating elaborate signaling networks across multiple cell-types; this makes it challenging to pinpoint therapeutically relevant drivers of IMDs. We developed an analytical framework which integrates Mendelian randomization (MR) and multiple-trait ...

Survivorship after Autologous Hematopoietic Cell Transplantation for Lymphoma and Multiple Myeloma: Late Effects and Quality of Life.

Although autologous hematopoietic cell transplantation (AHCT) is standard therapy for patients with lymphoma and multiple myeloma (MM), few studies have addressed late effects and quality of life (QOL) for long-term survivors after AHCT. Using long-term follow-up (LTFU) annual questionnaires with self-reported outcomes, we surveyed 665 patients who were ≥5 years after AHCT for the diagnosis of lymphoma or MM. Three-hundred and eighty-nine patients completed the questionnaire (58% response rate) a median o...


The Evolving Arena of Ovarian Cancer Maintenance Therapy.

Based on the results of several phase 3 randomized trials, "maintenance therapy" (prolonged treatment after an initial response to cytotoxic chemotherapy) has assumed a critical role in the routine care of advanced epithelial ovarian cancer. While earlier data had provided support for this therapeutic concept in disease management (e.g., multiple cycles of single-agent paclitaxel following a clinical complete response to a platinum/paclitaxel regimen), more recent data has revealed both the efficacy and saf...

One-lincRNA and five-mRNA based signature for prognosis of multiple myeloma patients undergoing proteasome inhibitors therapy.

Multiple myeloma is the second largest malignant tumor of the blood system. Proteasome inhibitors (PIs) currently are effective drugs for some myeloma patients, but their prognosis varies. We extracted the transcriptome expression data and clinical information of myeloma patients from MMRF CoMMpass database, and used the Random Survival Forest Variable Hunting (RSF-VH) algorithm to select 6 highly prognosis-related genes and to develop a 6-genes scoring model, by which the risk score predicted were signific...

Uveal melanoma: physiopathology and new in situ-specific therapies.

Uveal melanoma is the most common primary intraocular tumor in adults. It can arise from melanocytes in the anterior (iris) or posterior uveal tract (choroid and ciliary body). Uveal melanoma has a particular molecular pathogenesis, being characterized by specific chromosome alterations and gene mutations (e.g., GNAQ/GNA11; BAP1), which are considered promising targets for molecular therapy. Primary treatment of uveal melanoma includes radiotherapy (brachytherapy and charged-particle therapy), phototherapy ...

Simultaneous optimization of mixed photon energy beams in volumetric modulated arc therapy.

Despite the availability of multiple energy photon beams on clinical linear accelerators, volumetric modulated arc therapy (VMAT) optimization is currently limited to a single photon beam. The purpose of this work was to present a proof of principle study on an algorithm for simultaneous optimization of mixed photon beams for VMAT (MP - VMAT), utilizing an additional photon energy as an additional degree of freedom.

Overexpression of a trypanothione synthetase gene from Trypanosoma cruzi, TcTrys, confers enhanced tolerance to multiple abiotic stresses in rice.

Plants are frequently exposed to variable environmental stresses that adversely affect plant growth, development and agricultural production. In this study, a trypanothione synthetase gene from Trypanosoma cruzi, TcTryS, was chemically synthesized and its roles in tolerance to multiple abiotic stresses were functionally characterized by generating transgenic rice overexpressing TcTryS. Overexpression of TcTryS in rice endows transgenic plants with hypersensitivity to ABA, hyposensitivity to NaCl- and mannit...

Treatment Patterns and Clinical and Economic Outcomes in Patients With Newly Diagnosed Multiple Myeloma Treated With Lenalidomide- and/or Bortezomib-containing Regimens Without Stem Cell Transplant in a Real-world Setting.

Real-world data in patients with newly diagnosed multiple myeloma (NDMM) are sparse. Using United States claims databases, we analyzed treatment patterns, clinical outcomes, and health care utilization and costs in patients receiving lenalidomide- and/or bortezomib-containing therapy.

Biallelic mutations in Sperm flagellum 2 cause human multiple morphological abnormalities of the sperm flagella (MMAF) phenotype.

Male patients with multiple morphological abnormalities of the sperm flagella (MMAF) are infertile and exhibit absent, short, coiled, bent and/or irregular sperm flagella. Mutations in the SPEF2 gene reduce sperm motility and cause sperm tail defects in animal models and humans. In the present study, we performed a genetic analysis on an MMAF patient and identified novel biallelic mutations in the SPEF2 gene. The biallelic mutations were confirmed by Sanger sequencing, and in silico analysis revealed that t...

Stopping 5-Aminosalicylate Therapy in Patients With Crohn's Disease Starting Biologic Therapy Does Not Increase Risk of Adverse Outcomes.

Little is known about the effects of discontinuing 5-aminosalycilates (5-ASA) therapy for patients with Crohn's disease (CD) who initiate therapy with an anti-tumor necrosis factor (anti-TNF) agent. We analyzed data from 2 national population cohorts to compare outcomes of patients with CD already on 5-ASA therapy who started treatment with an anti-TNF agent and then either stopped or continued 5-ASA.

Photodynamic therapy for the treatment of oral squamous carcinoma - clinical implications resulting from in vitro research.

Oral cavity squamous cell carcinoma is a common cancer of the head and neck region. Due to the frequency of diagnoses, high rate of mortality, mutilating nature of classic therapy and numerous complications, new methods of treatment are being sought. One promising solution for treatment which is utilized in many fields of oncology is photodynamic therapy. The purpose of this article is to present a general overview of the use of photodynamic therapy in preclinical in vitro studies.

Phylogenomics of the genus Populus reveals extensive interspecific gene flow and balancing selection.

Phylogenetic analysis is complicated by interspecific gene flow and the presence of shared ancestral polymorphisms, particularly those maintained by balancing selection. In this study, we aimed to examine the prevalence of these factors during the diversification of Populus, a model tree genus in the Northern Hemisphere. We constructed phylogenetic trees of 29 Populus taxa using 80 individuals based on re-sequenced genomes. Our species tree analyses recovered four main clades in the genus based on consensus...

Engineering CRISPR mouse models of cancer.

Gene targeting in mammals has revolutionized the study of complex diseases, involving the interaction of multiple genes, cells, and organ systems. In cancer, genetically engineered mouse models deciphered biological principles by integrating molecular mechanisms, cellular processes, and environmental signals. Major advances in manipulative mouse genetics are currently emerging from breakthroughs in gene editing, which open new avenues for rapid model generation. Here, we review recent developments in engine...

Gene expression profiles of TNF-like cytokine 1A (TL1A) and its receptors death receptor 3 (DR3) and decoy receptor 3 (DcR3) in multiple sclerosis.

TL1A/DR3/DcR3 pathway is an important mediator of inflammatory responses and contributes to the pathogenesis of several chronic inflammatory diseases. Therefore, we analysed PBMC gene expression of these molecules in 30 relapsing-remitting multiple sclerosis (RRMS) patients, 8 secondary progressive MS (SPMS), 9 primary progressive MS (PPMS), 11 clinically isolated syndrome (CIS) patients, and 16 healthy controls (HCs), to evaluate their biomarker potential in MS. The results showed significant decrease in T...

Efficacy of proton therapy in children with high-risk and locally recurrent neuroblastoma.

Proton therapy is currently used in the management of pediatric tumors to decrease late toxicities. However, one of the criticisms of proton therapy is the limited data regarding efficacy on disease control. The purpose of this study was to examine local and distant control rates after proton therapy for neuroblastoma.

Transcriptome analysis of transcription factor genes under multiple abiotic stresses in Populus simonii × P.nigra.

Transcription factor (TF) genes play essential roles in abiotic stress responses as master switches in complex regulatory networks. In the present study, the transcript abundance of 4287 TF genes in Populus simonii × P.nigra were profiled under NaCl, KCl, CdCl and PEG stresses, respectively. A total of 118 up-regulated and 226 down-regulated TFs were identified to be shared in the four stress conditions. Among the top seven TF families (ERF, NAC, WRKY, MYB, bHLH, C2H2, bZIP), there were 76 up-regulated TFs...

Time-dependent Photodynamic Therapy for Multiple Targets: A Highly Efficient AIE-active Photosensitizer for Selective Bacterial Elimination and Cancer Cell Ablation.

Pathogen infection and cancer are the two major human health problems. In this work, we achieved an organic salt photosensitizer (PS), called 4TPA-BQ with aggregation-induced emission feature via one-step reaction. Owing to the aggregation-induced reactive oxygen species generation effect and sufficient small ΔE ST , 4TPA-BQ shows a satisfactorily high 1 O 2 generation efficiency of 97.8%. In vitro and in vivo experiments confirmed that 4TPA-BQ exhibited potent photodynamic antibacterial performance agains...

Conditional targeting in mice reveals that hepatic homogentisate 1,2-dioxygenase activity is essential in reducing circulating homogentisic acid and for effective therapy in the genetic disease alkaptonuria.

Alkaptonuria is an inherited disease caused by homogentisate 1,2-dioxygenase (HGD) deficiency. Circulating homogentisic acid (HGA) is elevated and deposits in connective tissues as ochronotic pigment. In this study, we aimed to define developmental and adult HGD tissue expression and determine the location and amount of gene activity required to lower circulating HGA and rescue the alkaptonuria phenotype. We generated an alkaptonuria mouse model using a knockout-first design for the disruption of the HGD ge...

Biomedical application of graphene: From drug delivery, tumor therapy, to theranostics.

The rise of graphene has driven great revolution in various fields, e. g. electronics, device, energy, etc., owing to the well-researched understanding of its physical and chemical properties. In the very recent decade, scientists have poured significant efforts to explore the biological functions of graphene and expand its biomedical applications, including drug delivery, tumor therapy, and theranostics. Encouraged by the inspiring research results and promising contributions of graphene to biomedicine fie...

Development of an interactive tumor vascular suppression strategy to inhibit multidrug resistance and metastasis with pH/HO responsive and oxygen-producing nanohybrids.

An ideal cancer therapeutic strategy should not only reverse multidrug resistance (MDR), but also prevent cancer metastasis. In this study, bovine serum albumin (BSA) was hybridized with Mn2+via biomineralization to develop a hybrid protein oxygen nanocarrier, which contained doxorubicin (DOX) and small interfering RNA (siRNA). The nanohybrid has the function of producing oxygen and chemotherapy synergistic gene therapy. FA-BSA-MnO2/DOX/siRNA was favorable for increasing the sensitivity of MCF-7/ADR cells t...

A comprehensive, multidisciplinary, precision medicine approach to discover effective therapy for an undiagnosed, progressive, fibroinflammatory disease.

Precision medicine has generated diagnoses for many patients with challenging undiagnosed disorders. Some individuals remain without a diagnosis despite comprehensive testing, and this impedes their treatment. This report addresses the role of personalized medicine in identifying effective therapy for an undiagnosed disease. A 22-year-old woman presented with chronic severe recurrent trismus, facial pain, progressive multicentric inflammatory and fibrotic masses, and high C-reactive protein. Sites of diseas...

Fingolimod for the treatment of multiple sclerosis in French West Indies, a real-world study in patients from African ancestry.

Affecting 2.5 million people worldwide, multiple sclerosis (MS) is one of the main causes of acquired disability among young adults. In French West Indies (FWI), where MS has arisen from the end of the 80's, a low therapeutic response to interferons beta1 (IFN beta1) in patients of African descent, restrains the therapeutic options. Fingolimod is a Sphingosine-1-Phosphate receptor modulator whose efficacy in the treatment of MS has recently been shown in three phase III studies. Nevertheless, data are curre...

Gene regulation analysis of the effects of evodiamine on tongue squamous cell carcinoma.

To use gene chip technology to study the effects of evodiamine (EVO) on the gene expression profile of tongue squamous cell carcinoma (TSCC) CAL-27 cell line, for the purpose of analyzing the mechanisms underlying the effects of EVO on gene expression and functional regulation of TSCC cells at the gene level.

Transcriptome and Regulatory Network Analyses of CD19-CAR-T Immunotherapy for B-ALL.

Chimeric antigen receptor (CAR) T-cell therapy has exhibited dramatic anti-tumor efficacy in clinical trials. In this study, we reported the transcriptome profiles of bone marrow cells in four B-cell acute lymphoblastic leukemia (B-ALL) patients before and after CD19-specific CAR-T therapy. CD19-CAR-T therapy remarkably reduced the number of leukemia cells, and three patients achieved bone marrow remission (minimal residual disease negative). The efficacy of CD19-CAR-T therapy on B-ALL was positively correl...


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