PubMed Journals Articles About "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" RSS

11:27 EDT 21st October 2019 | BioPortfolio

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Showing "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" PubMed Articles 1–25 of 31,000+

Hemophilia Gene Therapy: From Bench to Bed Side.

Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia.

ERT Degrades Gene Therapy for Storage Disorder.

Cycling at the Frontiers of Gene Therapy.

Sir Patrick Vallance is Government Chief Scientific Adviser in the United Kingdom. Here he discusses his path from academia to industry to government, and he reflects on the crucial early conversations that were instrumental in positioning gene therapy research for successful clinical development.

Basic concepts, current evidence, and future potential for gene therapy in managing cutaneous wounds.

Several studies have investigated the role of gene therapy in the healing process. The aim of this review is to explain the gene delivery systems in wound area.

Biosimilar Gene Therapy: Investigational Assessment of Secukinumab Gene Therapy.

Tumor necrosis factor-alpha (TNF-α), checkpoint inhibitors, and interleukin-17 (IL-17) are critical targets in inflammation and autoimmune diseases. Monoclonal antibodies (mAbs) have a successful portfolio in the treatment of chronic diseases. With the current progress in stem cells and gene therapy technologies, there is the promise of replacing costly mAbs production in bioreactors with a more direct and cost-effective production method inside the patient's cells. In this paper we examine the results of ...

Comparative Stability Studies of Different Infliximab and Biosimilar CT-P13 Clinical Solutions by Combined Use of Physicochemical Analytical Techniques and Enzyme-Linked Immunosorbent Assay (ELISA).

There are two products in which infliximab is the active pharmaceutical ingredient. These are Remicade (INF; reference product) and Remsima™/Inflectra™ (CT-P13; infliximab biosimilar). Remsima™/Inflectra™ are bioidentical products. Different recommendations have been made for the clinical solutions of each brand (Remicade or Remsima™/Inflectra™) despite the manufacturer of the biosimilar claiming high levels of similarity to the innovator.

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.

Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has be...

Gene therapy research for kidney diseases.

A resurgence in the development of newer gene therapy systems has led to recent successes in the treatment of B-cell cancers, retinal degeneration and neuromuscular atrophy. Gene therapy offers the ability to treat the patient at the root cause of their malady by restoring normal gene function and arresting the pathological progression of their genetic disease. The current standard of care for most genetic diseases is based upon the symptomatic treatment using polypharmacy while minimizing any potential adv...

Carbon nanotube-delivered iC9 suicide gene therapy for killing breast cancer cells in vitro.

To induce a safe death to MCF-7 human breast cancer cell line through gene therapy based on iC9 suicide gene.

Gene Therapy Briefs.

Gene Therapy Briefs.

DNAzyme-Loading MOFs for Self-Sufficient Gene Therapy.

DNAzymes have been recognized as potent therapeutic agents for gene therapy, while their inefficient intracellular delivery and insufficient cofactor supply precludes their practical biological applications. Metal-organic frameworks (MOFs) have emerged as promising drug carriers without in-depth consideration of their disassembled ingredients. Herein, we report a self-sufficient MOFs-sustained chlorin e6-modified DNAzyme (Ce6-DNAzyme) therapeutic nanosystem for combined gene therapy and photodynamic therapy...

Gene Therapy.

Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment. GTMPs to treat rare disorders caused by single-gene mutations have also made important advancements toward market availability, with eye and hematopoietic system diseases as the main applications.Nucleic acid-marketed pr...

Long-Term Safety and Efficacy of Gene-Pulmonary Macrophage Transplantation Therapy of PAP in Csf2ra Mice.

Hereditary pulmonary alveolar proteinosis (PAP) is a genetic lung disease characterized by surfactant accumulation and respiratory failure arising from disruption of GM-CSF signaling. While mutations in either CSF2RA or CSF2RB (encoding GM-CSF receptor α or β chains, respectively) can cause PAP, α chain mutations are responsible in most patients. Pulmonary macrophage transplantation (PMT) is a promising new cell therapy in development; however, no studies have evaluated this approach for hereditary PAP (...

Overcoming multiple drug resistance in lung cancer using siRNA targeted therapy.

Among cancers, lung cancer is the most morbidity and mortality disease that is remaining the fatalist. Generally, there are multiple treatment procedures for lung cancer, such as surgery, immunotherapy, radiotherapy and chemotherapy. There is, therefore, an urgent need for more specified and efficient methods for treatment of lung cancer such as RNAi, which in combination with traditional therapies could silence genes that are involved in the drug resistance. These genes may either be motivators of apoptosi...

Gene therapy of hematological disorders: current challenges.

Recent advances in genetic engineering technology and stem cell biology have spurred great interest in developing gene therapies for hereditary, as well as acquired hematological disorders. Currently, hematopoietic stem cell transplantation is used to cure disorders such as hemoglobinopathies and primary immunodeficiencies; however, this method is limited by the availability of immune-matched donors. Using autologous cells coupled with genome editing bypasses this limitation and therefore became the focus o...

Gene Delivery Using Lipoplexes and Polyplexes: Principles, Limitations and Solutions.

Gene therapy has attracted considerable attention for the treatment of genetic and acquired diseases. Successful gene therapy occurs when the therapeutic genes penetrate targeted cells and become available to the intracellular active site. Currently, a promising approach in gene delivery is the use of nonviral gene delivery vectors that lack immunogenicity but have low toxicity and potential tissue specificity. The widely used, existing nonviral gene vectors are cationic lipids and polymers that can pass ac...

Letter to the Editors: Re: Noninvasive Tests in Advanced Fibrosis Due to NASH: Critical Insights From STELLAR Trials.

The recently published article by Anstee et al. provides key insights into the complex etiopathogenesis of nonalcoholic steatohepatitis (NASH)-mediated advanced fibrosis. STELLAR-trials baseline data strongly emphasized predictive-value(s)/reliability of non-invasive tests (NITs) for bias-free, accurate assessment of liver-fibrosis, thereby warranting future public health research for significantly diminishing the increasing burden of hepatic malignancies in susceptible populations worldwide. This article ...

Automation in cell and gene therapy manufacturing: from past to future.

As more and more cell and gene therapies are being developed and with the increasing number of regulatory approvals being obtained, there is an emerging and pressing need for industrial translation. Process efficiency, associated cost drivers and regulatory requirements are issues that need to be addressed before industrialisation of cell and gene therapies can be established. Automation has the potential to address these issues and pave the way towards commercialisation and mass production as it has been t...

Chitosan for gene delivery: Methods for improvement and applications.

Gene therapy is a promising strategy for treating challenging diseases. The successful delivery of genes is a critical step for gene therapy. However, concerns about immunogenicity and toxicity are the main obstacles against the widespread use of effective viral systems. Therefore, nonviral vectors are regarded as good alternatives to viral vectors. Chitosan is a natural cationic polysaccharide that could be used to create nonviral gene delivery vectors. Various methods have been developed to improve the pr...

Down-regulation of taurine-up-regulated gene 1 attenuates inflammation by sponging miR-9-5p via targeting NF-κB1/p50 in multiple sclerosis.

Multiple sclerosis (MS) is an inflammatory disease of the central nervous system characterized by widespread inflammation. LncRNA taurine-up-regulated gene 1 (TUG1) has been reported to be involved in multiple biological processes and human diseases. The aim of this study was to investigate the role of lncRNA TUG1 in MS and the underlying mechanism.

Synchronous multiple myeloma and Gaucher disease.

Pseudo-Gaucher cells can be found in multiple hematologic malignancies, hemoglobinopathies, infections, and multiple storage disorders upon bone marrow aspirate and biopsy; however, Gaucher disease (GD) should be ruled out, particularly when the cytoplasmic inclusions cannot be adequately characterized. It is well known that GD may be associated with monoclonal gammopathies; however, although enzyme replacement therapy (ERT) may result in an improvement in polyclonal gammopathies, its effect on the progress...

B cell depletion therapy resulting in sustained remission of severe autoimmune complications following Alemtuzumab treatment of Multiple Sclerosis.

Secondary autoimmune disorders (AID) are a recognised complication of alemtuzumab treatment for multiple sclerosis. We have previously reported two female multiple sclerosis patients treated with alemtuzumab who developed rare but severe secondary AID; acquired haemophilia A and autoimmune encephalitis with seizures. Both cases proved to be refractory to treatment with conventional immuno-therapy. However, treatment of the patients with anti-CD20 therapy resulted in sustained remission. This observation val...

Discontinuation of disease-modifying therapy for patients with relapsing-remitting multiple sclerosis: Effect on clinical and MRI outcomes.

Disease-modifying therapy (DMT) for patients with relapsing-remitting multiple sclerosis (RRMS) have been shown to reduce relapses and new MRI lesions. However, few studies have assessed the impact of discontinuing DMT after a period of disease inactivity.

Gene therapy for refractory angina and cell therapy for heart failure: experience of a Brazilian research group.

Cell therapy has shown impressive effects in experimental cardiomyopathy models. To a lesser extent, gene therapy has also been studied. In both cases, translation to clinical therapy has been disappointing. This paper is intended to describe the experience and achievements of a multicenter working group located in Porto Alegre, southern Brazil, in experimental and translational research projects for cell-based and gene therapy methods in the treatment of dilated and ischemic cardiomyopathies. The results o...

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