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PubMed Journals Articles About "Agilis Biotherapeutics Gene Therapy Research Institution Enter Into" RSS

18:58 EST 17th February 2018 | BioPortfolio

Agilis Biotherapeutics Gene Therapy Research Institution Enter Into PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Agilis Biotherapeutics Gene Therapy Research Institution Enter Into articles that have been published worldwide.

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Showing "Agilis Biotherapeutics Gene Therapy Research Institution Enter into" PubMed Articles 1–25 of 43,000+

Is CAR-T Really Putting Us On Road to Gene Therapy?

CAR-T therapy involves some genetic engineering that is amazing; the culmination of work that began decades ago. But CAR T-cell therapy is not the classic form of gene therapy. Some refer to it as gene transfer that infuses copies of a normal gene or a modified gene into a genome in a more or less random fashion.


The Tetracycline Responsive System.

Constitutive gene expression is not always the appropriate expression system because the unphysiological levels of expressed protein could be detrimental in studies examining biological roles of proteins, or continued expression may be unnecessary after therapeutic effects have been achieved in gene therapy . We have utilized pharmacologically regulated gene expression systems to achieve fine control of gene expression levels which facilitate research in basic biology and translates to use in experimental g...

Gene therapy - from idea to reality Gene therapy was originally proposed 45 years ago, but it is only during the last 5-10 years that significant clinical benefit has been demonstrated. Gene therapy is in most cases in the form of engineered viruses carrying a therapeutic gene. Examples of successfully treated disorders are primary immunodeficiencies and hemophilias. In some cases, gene therapy consists of genetically modified cells, such as when chimeric antigen receptors are stably introduced into T lymph...


Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.

Malaysian researchers talk about the influence of culture on research misconduct in higher learning institutions.

Based on a previous survey by the Office of Research Integrity (ORI) in the USA, quite a number of foreign research scientists have been found guilty of research misconduct. However, it remains unclear as to whether or not cultural factors really contribute to research misconduct. This study is based on a series of interviews with Malaysian researchers from the local universities regarding their own professional experiences involving working with researchers or research students from different countries or ...

Pressing diseases that represent promising targets for gene therapy.

Over time, there has been a growing interest in the application of gene therapy within the healthcare industry as demonstrated by the nearly 3,000 clinical trials associated with gene therapy that are listed in clinicaltrials.gov. However, there are various difficulties associated with gene therapy that have limited the realization of licensed gene therapies to only a handful of treatments. Furthermore, efforts to develop gene therapeutics have been narrowly focused and most clinical trials have sought to d...

Gene therapy: evidence, value and affordability in the US health care system.

To explore the challenges presented by gene therapies, discuss potential solutions, and present policy recommendations.

Fight fire with fire: Gene therapy strategies to cure HIV.

Human Immunodeficiency Virus (HIV) to date remains one of the most notorious viruses mankind has ever faced. Despite enormous investments in HIV research for more than 30 years an effective cure for HIV has been elusive. Areas covered: Combination antiretroviral therapy (cART) suppresses active viral replication, but is not able to eliminate the virus completely due to stable integration of HIV inside the host genome of infected cells and the establishment of a latent reservoir, that is insensitive to cART....

FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B.

A phase 1/2 clinical trial of AAV-mediated gene therapy in patients with hemophilia B using an enhanced specific activity factor IX (FIX) transgene reports sustained levels of FIX levels, leading to the near elimination of bleeding for more than a year and without serious adverse side effects. These results are the best outcome to date for hemophilia gene therapy.

Compilation of online resources of relevance to 'Spinraza and advanced therapies: a stakeholder special' issue of Gene Therapy.

Integrating Spirituality into MFT Training: A Reflexive Curriculum and Qualitative Evaluation.

When clients enter the therapy room they bring unique spiritual beliefs and values in with them; however, marital and family therapists (MFTs) often lack training to assist in this important area of diversity. Current training recommendations encourage therapists to explore their own spirituality to identify personal limitations that may occlude positive client outcomes; however very little information exists on the implementation and usefulness of this type of training. This article will describe a curricu...

Carl June Speaks of His Pioneering Efforts That Led to the First Food and Drug Administration-Approved Gene Therapy Product.

Chemical modifications of nucleic acid drugs and their delivery systems for gene-based therapy.

Gene-based therapy is one of essential therapeutic strategies for precision medicine through targeting specific genes in specific cells of target tissues. However, there still exist many problems that need to be solved, such as safety, stability, selectivity, delivery, as well as immunity. Currently, the key challenges of gene-based therapy for clinical potential applications are the safe and effective nucleic acid drugs as well as their safe and efficient gene delivery systems. In this review, we first foc...

FDA panel recommends approval of first gene therapy in US.

The Renaissance of Gene and Cell Therapy: Florence 2016.

Disease characteristics and clinical outcomes in patients aged less than 40 with chronic lymphocytic leukemia.

Outcomes in very young CLL patients (age ≤40) are not well characterized. We compared 71 consecutive patients aged ≤40 with 142 "older" matched patients >40 from our institution and used SEER database as an independent comparison group. Patients in the two age groups were diagnosed at similar Rai stage. At diagnosis, very young patients had a similar rate of adverse cytogenetics, IGHV mutation and ZAP70 expression and had lower beta-2-microglobulin and a lower incidence of second malignancies. There was...

Gene therapy for heart failure: status quo and quo vadis.

Gene therapy is recently attracting increased attention and cardiac gene therapy is not an exception. Advances in gene transfer vectors, development of new vector delivery methods, and discovery of new gene targets continue to fuel our motivation to use this approach in routine bedside care. In the past two years, we have witnessed important advances in the field, as the results of three recently completed cardiac gene therapy programs have been published. Unfortunately, none of the trials have met their pr...

Genetic therapies for cystic fibrosis lung disease.

Gene therapy for cystic fibrosis (CF) has been the subject of intense research over the last twenty-five years or more, using both viral and liposomal delivery methods, but so far without the emergence of a clinical therapy. New approaches to CF gene therapy involving recent improvements to vector systems, both viral and non-viral, as well as new nucleic acid technologies have led to renewed interest in the field. The field of therapeutic gene editing is rapidly developing with the emergence of CRISPR/Cas9 ...

Gene therapy for Cystic Fibrosis: Improved delivery techniques and conditioning with lysophosphatidylcholine enhance lentiviral gene transfer in mouse lung airways.

Purpose/Aim: Cystic fibrosis (CF) is the most common, fatal recessive genetic disease among the Caucasian population. Gene therapy has the potential to treat CF long term, however physiological barriers can prevent VSV-G pseudotyped lentiviral (LV) vectors from efficiently accessing the relevant receptors on the basolateral membrane of airway epithelial cells. The aims of this experiment were to use our new dose delivery techniques to determine whether conditioning the mouse lung conducting airways with lys...

Collaborative Orthopaedic Research Between and Within Institutions.

Collaborative research is common in many medical disciplines; however, the field of orthopaedics has been relatively slow to adopt this type of research approach. Collaborative research efforts can occur between multiple institutions and, in some instances, may benefit from subspecialty society sponsorship. Collaborative research efforts between several research spheres within a single institution also can be advantageous. Collaborative research has many benefits, including a larger number of patients in st...

Gene Therapy Briefs.

Gene and Cell Therapy in 2018: A Look Ahead.

Gene Therapy for Alcoholism and Other Substance Use Disorders.

Gene Therapy Briefs.

A visual guide to gene/optothermal synergy therapy nanosystem using tungsten oxide.

Combination of gene therapy and photothermal therapy (PTT) has drawn much attention in cancer therapy in recent years. However, this joint treatment process lacks fluorescence imaging visualization guidance that limits its clinical applications in oncotherapy. Herein, we report the use of gene therapy and tungsten oxide (W18O49, WO) synthetized with template method for combined PTT of cancer. In this system, a novel nanoplatform, with Bax gene, WO and indocyanine green (ICG) loaded in mesoporous silica nano...


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