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PubMed Journals Articles About "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" - Page: 5 RSS

04:44 EST 17th 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 101–125 of 31,000+

Promising approaches of small interfering RNAs (siRNAs) mediated cancer gene therapy.

RNA interference (RNAi) has extensive potential to revolutionize every aspect of clinical application in biomedical research. One of the promising tools is the Small interfering RNA (siRNA) molecules within a cellular component. Principally, siRNA mediated innovative advances are increasing rapidly in support of cancer diagnosis and therapeutic purposes. Conversely, it has some delivery challenges to the site of action within the cells of a target organ, due to the progress of nucleic acids engineering and ...


NutriGenomeDB: a nutrigenomics exploratory and analytical platform.

Habitual consumption of certain foods has shown beneficial and protective effects against multiple chronic diseases. However, it is not clear by which molecular mechanisms they may exert their beneficial effects. Multiple -omic experiments available in public databases have generated gene expression data following the treatment of human cells with different food nutrients and bioactive compounds. Exploration of such data in an integrative manner offers excellent possibilities for gaining insights into the m...

Ex vivo gene therapy using human bone marrow cells overexpressing BMP-2: "Next-day" gene therapy versus standard "two-step" approach.

Traditionally, ex vivo gene therapy involves a two-step approach, with culture expansion of cells prior to transduction and implantation. We have tried to simplify this strategy and eliminate the time and cost associated with culture expansion, by introducing "next-day" regional gene therapy using human bone marrow cells. The purpose of this study was to determine whether a lentiviral vector (LV) carrying the cDNA for BMP-2 can transduce freshly isolated human BM cells, leading to abundant BMP production an...


Neonatal diabetes caused by the heterozygous Pro1198Leu mutation in the ABCC8 gene in a male infant: six-year clinical course.

Neonatal diabetes is a rare disease, often caused by monogenic abnormality. Our male infant patient developed diabetic ketoacidosis at two months of age due to the heterozygous ABCC8 gene mutation (p.Pro1198Leu). After genetic diagnosis, insulin therapy was successfully transitioned to oral sulfonylurea therapy. For over six years, oral sulfonylurea therapy has been safe and effective and the required amount of sulfonylureas has progressively decreased. The mutation was transmitted in an autosomal-dominant ...

Intrathymic AAV gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.

Patients with T cell immunodeficiencies are generally treated by allogeneic hematopoietic stem cell transplantation but alternatives are needed for patients without matched donors. An innovative intrathymic (IT) gene therapy approach, directly targeting the thymus, may improve outcome.

Mutational analysis of renal angiomyolipoma associated with tuberous sclerosis complex and the outcome of short-term everolimus therapy.

To identify clinical characteristics and mutation spectra in Chinese patients with renal angiomyolipoma (AML) associated with the tuberous sclerosis complex (TSC, TSC-AML), examined the efficacy and safety of short-term everolimus therapy (12 weeks). We analyzed the frequency distribution of each TSC-related clinical feature and investigated gene mutations by genetic testing. Some subjects received everolimus for 12 weeks at a dose of 10 mg/day, and the efficacy and safety of short-term everolimus therapy...

Co-assembled Ca Alginate-Sulfate Nanoparticles for Intracellular Plasmid DNA Delivery.

Successful gene therapy requires the development of suitable carriers for the selective and efficient delivery of genes to specific target cells, with minimal toxicity. In this work, we present a non-viral vector for gene delivery composed of biocompatible materials, CaCl, plasmid DNA and the semi-synthetic anionic biopolymer alginate sulfate (AlgS), which spontaneously co-assembled to form nanoparticles (NPs). The NPs were characterized with a slightly anionic surface charge (Zeta potential [ζ] = -14 mV...

High Expression Levels of Long Noncoding RNA Small Nucleolar RNA Host Gene 18 and Semaphorin 5A Indicate Poor Prognosis in Multiple Myeloma.

The aim of this study was to detect the expression of long noncoding RNA small nucleolar RNA host gene 18 (SNHG18) andsemaphorin 5A (SEMA5A) genes in multiple myeloma (MM) patients and to explore the correlation of the expression of these genes with the clinical characteristics and prognosis of MM patients.

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.

Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy is given. In previous studies, autologous gene therapy with γ-retroviral vectors failed to reconstitute B-cell and NK-cell immunity and was complicated by vector-related leukemia.

A CRISPR/LbCas12a-based method for highly efficient multiplex gene editing in Physcomitrella patens.

Due to their high efficiency, specificity, and flexibility, programmable nucleases such as those of the CRISPR/Cas12a (Cpf1) system have greatly expanded the applicability of editing the genomes of various organisms. Genes from different gene families or genes with redundant functions in the same gene family can be examined by assembling multiple CRISPR RNAs (crRNAs) in a single vector. However, the activity and efficiency of CRISPR/Cas12a in the nonvascular plant Physcomitrella patens are largely unknown. ...

Multiple genetic mutations implicate spectrum of phenotypes in Bardet-Biedl syndrome.

Bardet-Biedl syndrome (BBS) is a clinically and genetically heterogeneous ciliopathy with several clinical features including retinitis pigmentosa, obesity, kidney dysfunction, postaxial polydactyly, behavioral dysfunction and hypogonadism with wide spectrum of additional features. With multiple phenotypes and heterogeneous distribution, it is unlikely that BBS is caused by single gene defect. We have performed clinical and genetic diagnosis of two individuals from an Indian family with classical BBS sympto...

RNA-based Therapy for Osteogenesis.

Nucleic acid-based therapy has shown great promise in accelerating bone regeneration as well as other diseases. Nucleic acids used in gene therapy mainly are either plasmid DNA (pDNA) or RNAs. Although pDNA therapy has been extensively studied for decades with encouraging preclinical and clinical results, side effects, and low efficiency associated with nuclear trafficking are hard to bypass. Unlike pDNA, RNAs (mRNA, siRNA, miRNA) exert their function in the cytoplasm, thereby being more efficient in hard-t...

Mechanisms of acquired tumor drug resistance.

Systemic therapy often results in the reduction of tumor size but rarely succeeds in eradicating all cancer cells. Drug efflux, persistence of cancer stem cells (CSCs), epithelial-mesenchymal transition (EMT) and down-regulation of apoptosis are the most known general causes of therapy failure. Tumor escape from targeted compounds often involves pathway-specific mechanisms, which result in the restoration of the affected signaling cascade. The acquisition of drug resistance is mediated by mutations, changes...

Prospective evaluation of insulin-to-carbohydrate ratio in children and adolescents with type 1 diabetes using multiple daily injection therapy.

Assessment of insulin-to-carbohydrate ratio (ICR) in children and adolescents with type 1 diabetes mellitus (T1DM) using multiple daily injection (MDI) therapy.

Star-shaped poly(2-aminoethyl methacrylate)s as non-viral gene carriers: Exploring structure-function relationship.

Gene therapy shows much promise in treating many inheritable and acquired diseases, but challenges remain in the design of gene vectors with low cytotoxicity and high transfection efficiency. Elucidating the structure-function relationship of non-viral polymer-based gene carriers is crucial for improving the design and performance of safe and effective gene therapy approaches. The cationic poly(2-aminoethyl methacrylate) (PAEM) containing primary amino side groups is an attractive carrier for gene delivery....

Utilizing multiplex fluor LAMPs to illuminate multiple gene expressions in situ.

In situ gene expression detection is the best way to determine temporal and spatial differences in gene expression. However, in situ hybridization procedures are inherently difficult to execute and typically suffer from degradation of sample tissues, limited sensitivity to genes with low expression, high background, and limitation to single gene detections. We propose to utilize an isothermal gene amplification technique, LAMP (Loop-Mediated Isothermal Amplification), to solve these problems in a novel way....

Gene ontology analysis of arthrogryposis (multiple congenital contractures).

In 2016, we published an article applying Gene Ontology Analysis to the genes that had been reported to be associated with arthrogryposis (multiple congenital contractures) (Hall & Kiefer, 2016). At that time, 320 genes had been reported to have mutations associated with arthrogryposis. All were associated with decreased fetal movement. These 320 genes were analyzed by biological process and cellular component categories, and yielded 22 distinct groupings. Since that time, another 82 additional genes have b...

Epigenetically Upregulated MicroRNA-602 Is Involved in a Negative Feedback Loop with FOXK2 in Esophageal Squamous Cell Carcinoma.

MicroRNA is an endogenous, small RNA controlling multiple target genes and playing roles in various tumorigenesis processes. In this study, our results revealed that miR-602 expression levels in tumor tissues and preoperative serum from esophageal squamous cell carcinoma (ESCC) patients were higher than those in non-tumorous tissues and healthy volunteers. miR-602 overexpression was closely related to lymph node metastasis and TNM stages and correlated short overall, and it acted as an independent prognosti...

Smart Cell-Specific Protein Therapeutics for Head and Neck Cancer.

Gene expression through protein delivery is a safer alternative to gene therapy vectors as rapid, robust, and short-term gene expression can be achieved without the accompanying risk of insertional mutagenesis. Cell-penetrating peptides (CPPs) are a diverse class of peptides that have been derived from viral proteins or developed synthetically. These peptides can readily translocate into live cells in vitro and in vivo, and when fused to proteins, they are an invaluable tool for protein internalization and ...

A versatile toolbox for knock-in gene targeting based on the Multisite Gateway technology.

Knock-in (KI) gene targeting can be employed for a wide range of applications in stem cell research. However, vectors for KI require multiple complicated processes for construction, including multiple times of digestion/ligation steps and extensive restriction mapping, which has imposed limitations for the robust applicability of KI gene targeting. To circumvent this issue, here we introduce versatile and systematic methods for generating KI vectors by molecular cloning. In this approach, we employed the Mu...

Optimization of peptide-plasmid DNA vectors formulation for gene delivery in cancer therapy exploring design of experiments.

The field of gene therapy still attracts great interest due to its potential therapeutic effect towards the most deadly diseases, such as cancer. For cancer gene therapy to be feasible and viable in a clinical setting, the design and development of a suitable gene delivery system is imperative. Peptide based vectors, in particular, reveal to be promising for therapeutic gene release. Following this, two different peptides, RALA and WRAP5, have been investigated mainly regarding their ability to form complex...

DNA methylation analysis of negative pressure therapy effect in diabetic foot ulcers.

Negative pressure wound therapy (NPWT) has been used to treat diabetic foot ulcerations (DFUs). Its action on the molecular level, however, is only partially understood. Some earlier data suggested NPWT may be mediated through modification of local gene expression. As methylation is a key epigenetic regulatory mechanism of gene expression, we assessed the effect of NPWT on its profile in patients with type 2 diabetes (T2DM) and neuropathic non-infected DFUs.

Challenges of Gene Delivery to the Central Nervous System and the Growing use of Biomaterial Vectors.

Gene therapy is a promising form of treatment for those suffering from neurological disorders or central nervous system (CNS) injury, however, obstacles remain that limit its translational potential. The CNS is protected by the blood brain barrier, and this barrier blocks genes from traversing into the CNS if administered outside of the CNS. Viral and non-viral gene delivery vehicles, commonly referred to as vectors, are modified to enhance delivery efficiency to target locations in the CNS. Still, there ar...

The elevated level of osteopontin in patients with pemphigus vulgaris: a cytokine-like protein with a therapeutic potential.

Pemphigus is a life-threatening autoimmune disease with no certain treatment. Anticytokine therapy is being increasingly discussed in multiple autoimmune diseases. Osteopontin (OPN) is a glycoprotein protein produced by a variety of immune cells. Increased OPN serum levels have been reported in several autoimmune diseases, with targeting OPN considered as a promising therapy in these diseases. However, the role of OPN in PV has not been well studied so far.

Biologic therapy in the idiopathic inflammatory myopathies.

The idiopathic inflammatory myopathies (IIM) are a group of autoimmune diseases resulting from inflammation of muscle and manifesting as weakness, though a range of extra-muscular manifestations are observed. These are often correlated closely with disease subtype and the presence of myositis-specific/myositis-associated antibodies. IIM are notoriously difficult to treat and often refractory to glucocorticoid therapy and synthetic immunosuppressants. Both the innate and adaptive immune systems are implicate...


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