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01:38 EDT 25th May 2018 | BioPortfolio

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Showing "Agilis Biotherapeutics Gene Therapy Research Institution Enter into" PubMed Articles 1–25 of 44,000+

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.

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 ...

Radiation Gene-expression Signatures in Primary Breast Cancer Cells.

In breast cancer (BC) care, radiation therapy (RT) is an efficient treatment to control localized tumor. Radiobiological research is needed to understand molecular differences that affect radiosensitivity of different tumor subtypes and the response variability. The aim of this study was to analyze gene expression profiling (GEP) in primary BC cells following irradiation with doses of 9 Gy and 23 Gy delivered by intraoperative electron radiation therapy (IOERT) in order to define gene signatures of response...

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 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.

Data collection of patient outcomes: one institution's experience.

Patient- and provider-reported outcomes are recognized as important in evaluating quality of care, guiding health care policy, comparative effectiveness research, and decision-making in radiation oncology. Combining patient and provider outcome data with a detailed description of disease and therapy is the basis for these analyses. We report on the combination of technical solutions and clinical process changes at our institution that were used in the collection and dissemination of this data. This initiati...

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...

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.

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

Effective gene therapy for hemophilia, at last….

Recent efforts at gene therapy for haemophilia A and B using AAV-derived vectors show durable expression of coagulation factors at significant levels, resulting in almost complete correction of the phenotype. This is the first success of gene therapy for a major hereditary disorder, and shows how continuous improvement of many components of the system has finally succeeded. Although these results must be confirmed with more patients and longer durations, they constitute a significant accomplishment for this...

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...

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...

Correction to: Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World, by Zhang J, Yan J, and Zeng F. Hum Gene Ther 2018;29(2):197-203. DOI: 10.1089/hum.2017.228.

The Renaissance of Gene and Cell Therapy: Florence 2016.

Visual Field Variability after Gene Therapy for Leber's Hereditary Optic Neuropathy.

To assess changes in visual field (VF) values after gene therapy for Leber's hereditary optic neuropathy (LHON).

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...

Gene therapy in optic nerve disease.

Highlight some of the recent advances in gene therapy and gene modification for optic nerve disease to promote axon regeneration, neuroprotection, and increased visual functioning.

Gene Therapy Briefs.

Gene and Cell Therapy in 2018: A Look Ahead.

Adipose Stem Cell Therapy for Chronic Pancreatitis.

Toward best practices in data processing and analysis for intact biotherapeutics by MS in quantitative bioanalysis.

Typically, quantitation of biotherapeutics from biological matrices by LC-MS is based on a surrogate peptide approach to determine molecule concentration. Recent efforts have focused on quantitation of the intact protein molecules or larger mass subunits of monoclonal antibodies. To date, there has been limited guidance for large or intact protein mass quantitation for quantitative bioanalysis.

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