Topics

PubMed Journals Articles About "Non-Viral TCR Gene Therapy" RSS

06:17 EST 24th January 2020 | BioPortfolio

Non-Viral TCR Gene Therapy PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Non-Viral TCR Gene Therapy articles that have been published worldwide.

More Information about "Non-Viral TCR Gene Therapy" on BioPortfolio

We have published hundreds of Non-Viral TCR Gene Therapy news stories on BioPortfolio along with dozens of Non-Viral TCR Gene Therapy Clinical Trials and PubMed Articles about Non-Viral TCR Gene Therapy for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Non-Viral TCR Gene Therapy Companies in our database. You can also find out about relevant Non-Viral TCR Gene Therapy Drugs and Medications on this site too.

Showing "Viral Gene Therapy" PubMed Articles 1–25 of 24,000+

Immune Responses to Viral Gene Therapy Vectors.

Several viral vector-based gene therapy drugs have now received marketing approval. A much larger number of additional viral vectors are in various stages of clinical trials for the treatment of genetic and acquired diseases, with many more in pre-clinical testing. Efficiency of gene transfer and ability to provide long-term therapy make these vector systems very attractive. In fact, viral vector gene therapy has been able to treat or even cure diseases for which there had been no or only suboptimal treatme...


Gene therapy for severe combined immunodeficiencies and beyond.

Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase (ADA) deficiency. The rationale was brought up by the observation of the revertant of SCIDX1 and ADA deficiency as a kind of natural gene therapy. Nevertheless, the first attempts of gene therapy for SCID X1 were associated with insertional mutagenesis causing leukemia, because the viral enhancer induced transactivation of oncogenes...

Emerging gene therapies for cystic fibrosis.

: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few clinically effective treatment options. Gene therapy and gene editing strategies offer the potential for a one-time CF cure, irrespective of the mutation class. : We review emerging gene therapies and gene delivery strategies for the treatment of CF particularly viral and non-viral approaches with potential to treat CF. : It was initially anticipated that the challenge of developing a gene therapy for CF lung disease would be met r...


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.

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...

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....

Adeno-Associated Virus as Gene Delivery Vehicle into the Retina.

Initially discovered as a contaminant of adenovirus preparations, adeno-associated virus (AAV) has proved one of the most promising viral vectors for human gene therapy. The safety profile of AAV has been well-characterized in vivo studies, and the first gene therapy for patients with vision loss caused by Leber congenital amaurosis or retinitis pigmentosa was approved by the US Food and Drug Administration in 2017. This is an exciting era for investigators working on retina biology and treatments for blind...

The National Gene Vector Biorepository. Eleven Years of Providing Resources to the Gene Therapy Community.

The NGVB program has been highly accessed by gene therapy investigators. The Reagent Repository has distributed over 1,000 reagents to 397 investigators. The Pharmacology/Toxicology Archive contains over 36,000 specimens from a variety of AAV, Adenoviral and other Pharmacology/Toxicology studies. NGVB also maintains a searchable database of Gene Therapy Pharmacology/Toxicology studies to promote data sharing. NGVB has provided FDA mandated replication competent virus testing for over 70 clinical trials. Fro...

Immune response and gene therapy with adenoassociated viral vectors.

In recent years, gene therapy has been positioned as a real and safe option in the development of therapeutic alternatives for the cure and prevention of different diseases. It consists in the insertion of genetic material in a defective tissue or cell, through the use of a vector. There are several considerations for selecting the most appropriate vector, including the potential for binding and entry to the target cell, the ability of the genetic material to transfer to the nucleus, the ability to express ...

ERT Degrades Gene Therapy for Storage Disorder.

TRAIL-based gene delivery and therapeutic strategies.

TRAIL (tumor necrosis factor-related apoptosis-inducing ligand), also known as APO2L, belongs to the tumor necrosis factor family. By binding to the death receptor 4 (DR4) or DR5, TRAIL induces apoptosis of tumor cells without causing side toxicity in normal tissues. In recent years TRAIL-based therapy has attracted great attention for its promise of serving as a cancer drug candidate. However, the treatment efficacy of TRAIL protein was under expectation in the clinical trials because of the short half-lif...

Ligand targeting and peptide functionalized polymers as non-viral carriers for gene therapy.

Polymeric gene carriers have been developed to deliver therapeutic genes for treating various genetic diseases. They may diminish the problems related with viral vectors in terms of safety, immunogenicity and mutagenesis. But inefficient endosomal release, cytoplasmic transport and nuclear entry are the main limiting issues in the usage of polymeric carriers. Different strategies have been proposed to functionalize gene carriers for individually overcoming these barriers. Towards this aim, various polymeric...

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.

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.

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 ...

Cholesterol-conjugated PEGylated PAMAM as an Efficient Nanocarrier for Plasmid Encoding Interleukin-12 Immunogene Delivery towards Colon Cancer Cells.

IL-12 is a pleiotropic cytokine, which shows an ideal applicant for tumour immunotherapy, because of its features of creating an interconnection between innate (NK cells) and adaptive (cytotoxic T lymphocyte) immunity. IL-12 gene therapy is a useful technique to deliver an immune-modulatory gene directly into tumour site thereby limiting the adverse effects of systemic administration of IL-12 proteins. One of the most largely investigated non-viral gene carriers is Polyamidoamine (PAMAM). In the current res...

Dry AMD - Cellular and Genetic Therapies.

The growing incidence of neurodegenerative diseases is based on our increasingly aging society as well as the difficulties in establishing defined therapy regimens. For dry age-related macular degeneration (AMD) and the later stage of geographic atrophy (GA), various treatment options exist that only decelerate the progression of the disease. However, no therapy is currently available that can restore the degenerated retinal pigment epithelium (RPE) and/or photoreceptor cells. Cellular and gene-based approa...

The approved gene therapy drugs worldwide: from 1998 to 2019.

With the improvement of gene vectors, the rise of chimeric antigen receptor T cell immunotherapy and breakthroughs in the genome editing technology, gene therapy had once again returned to the central stage of disease treatment. It had brought new choices to clinical therapy of diseases such as tumors and genetic diseases, and had changed the status quo of treatment for monogenic disorders and diffuse large B-cell lymphoma. Until August 2019, 22 gene medicines had been approved by the drug regulatory agenci...

Multigene vector delivery with herpes simplex virus 1 amplicons.

Gene expression studies and gene therapy require efficient gene delivery into cells. Different technologies by viral and non-viral mechanisms have been used for gene delivery into cells. Small gene vectors transfer across the cell membrane with a relatively high efficiency, but not large genes or entire loci spanning several kilobases, which do not remain intact following introduction. Previously, we developed an efficient delivery system based on herpes virus simplex type 1 (HSV-1) amplicons to transfer la...

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 ...

i-Silence, Please! An Alternative for Gene Disruption via Adenine Base Editors.

Viral-mediated gene therapy and genetically modified therapeutics: A primer on biosafety handling for the health-system pharmacist.

The guidance documents applicable to the manipulation of viral vectors in a health-system pharmacy are reviewed to provide recommendations for occupational safe drug handling.

Encouraging and Unsettling Findings in Long-Term Follow-up of AAV Gene Transfer.

Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy.

Translational Medicine: Evolution, Fulfillment, and Belief in Gene Therapy.


Quick Search