PubMed Journals Articles About "Reader Inquiry Krystal Biotech Participating Rally Gene Therapy" RSS

04:01 EDT 22nd May 2018 | BioPortfolio

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Showing "Reader Inquiry Krystal Biotech Participating Rally Gene Therapy" PubMed Articles 1–25 of 23,000+

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.

BRD4 Promotes DNA Repair and Mediates the Formation of TMPRSS2-ERG Gene Rearrangements in Prostate Cancer.

BRD4 belongs to the bromodomain and extraterminal (BET) family of chromatin reader proteins that bind acetylated histones and regulate gene expression. Pharmacological inhibition of BRD4 by BET inhibitors (BETi) has indicated antitumor activity against multiple cancer types. We show that BRD4 is essential for the repair of DNA double-strand breaks (DSBs) and mediates the formation of oncogenic gene rearrangements by engaging the non-homologous end joining (NHEJ) pathway. Mechanistically, genome-wide DNA br...

Biotech Crop Planting Resumes High Adoption in 2016.

The global area of biotech crops in 2016 increased from 179.7 million hectares to 185.1 million hectares, a 3% increase equivalent to 5.4 million hectares. Some 26 countries planted biotech crops, 19 of which were developing countries and seven were industrial. Information and data collected from various credible sources showed variations from the previous year. Fluctuations in biotech crop area (both increases and decreases) are influenced by factors including, among others, acceptance and commercializatio...

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...

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.

Mechanisms of Change for Children Participating in Therapeutic Horse Riding: A Grounded Theory.

To develop a model for understanding mechanisms of change in health outcomes for riders with disabilities participating in therapeutic horse riding (THR).

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...

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.

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.

Targeted Interventions to Advance a Culture of Inquiry at a Large, Multicampus Hospital Among Nurses.

The aim of this study is to assist nurse leaders in developing innovative structures to foster a culture of inquiry among professional nurses.

Adipose Stem Cell Therapy for Chronic Pancreatitis.

Interleukins and their signaling pathways in the Reactome biological pathway database.

There is a wealth of biological pathway information available in the scientific literature but it is spread across many thousands of publications. Alongside publications that contain definitive experimental discoveries are many others that have been dismissed as spurious, or found to be irreproducible, or are contradicted by later results and consequently now considered controversial. Many descriptions and images of pathways are incomplete, stylized representations that assume the reader is an expert, famil...

Retinal gene therapy.

Inherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit.

High Resolution Melting Analysis for Rapid Detection of PIK3CA Gene Mutations in Bladder Cancer: A Mutated Target for Cancer Therapy.

PIK3CA gene mutations have clinical importance and their presence is associated with therapy response. They are also considered as a molecule for targeted therapy. As regards to their importance, genetic variation within a population as well as among different populations, this study was conducted to detect common mutations of exons 9 and 20 and other probable mutations in PIK3CA gene as well as their frequencies in Iranian bladder cancer patients.

Comparison of the editing patterns and editing efficiencies of TALEN and CRISPR-Cas9 when targeting the human CCR5 gene.

The human C-C chemokine receptor type-5 (CCR5) is the major transmembrane co-receptor that mediates HIV-1 entry into target CD4+ cells. Gene therapy to knock-out the CCR5 gene has shown encouraging results in providing a functional cure for HIV-1 infection. In gene therapy strategies, the initial region of the CCR5 gene is a hotspot for producing functional gene knock-out. Such target gene editing can be done using programmable endonucleases such as transcription activator-like effector nucleases (TALEN) or...

Relationship between Roe and Metz simulation model for multireader diagnostic data and Obuchowski-Rockette model parameters.

For the typical diagnostic radiology study design, each case (ie, patient) undergoes several diagnostic tests (or modalities) and the resulting images are interpreted by several readers. Often, each reader is asked to assign a confidence-of-disease rating to each case for each test, and the diagnostic tests are compared with respect to reader-performance outcomes that are functions of the reader receiver operating characteristic (ROC) curves, such as the area under the ROC curve. These reader-performance ou...

Nanoparticles for death‑induced gene therapy in cancer (Review).

Due to the high toxicity and side effects of the use of traditional chemotherapy in cancer, scientists are working on the development of alternative therapeutic technologies. An example of this is the use of death‑induced gene therapy. This therapy consists of the killing of tumor cells via transfection with plasmid DNA (pDNA) that contains a gene which produces a protein that results in the apoptosis of cancerous cells. The cell death is caused by the direct activation of apoptosis (apoptosis‑induced g...

Gene Therapy with the Sleeping Beauty Transposon System.

The widespread clinical implementation of gene therapy requires the ability to stably integrate genetic information through gene transfer vectors in a safe, effective, and economical manner. The latest generation of Sleeping Beauty (SB) transposon vectors fulfills these requirements, and may overcome limitations associated with viral gene transfer vectors and transient nonviral gene delivery approaches that are prevalent in ongoing clinical trials. The SB system enables high-level stable gene transfer and s...

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