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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.
This article was published in the following journal.
Name: Human gene therapy. Clinical development
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 hemo...
Methanethiol (MeSH) and dimethylsulfide (DMS) are volatile organic sulfur compounds (VOSCs) with important roles in sulfur cycling, signaling and atmospheric chemistry. DMS can be produced from MeSH t...
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 participation rates in Australia and Queensland remain low. Rural residents typically do not have distinct cycling infrastructure available for use. Investigating current cycling participation...
Exercise has been the cornerstone of diabetes management. However, many diabetic patients have ADL disabilities and experience substantial difficulty in performing usual exercises, such as...
The purpose of the trial is to evaluate the safety and efficacy of the Ablation Frontiers Cardiac Ablation System compared to currently approved treatment options for atrial fibrillation.
This study will seek to determine the benefits that FES-LE cycling has over cycling alone on walking performance and quality of life in people with multiple sclerosis.
The purpose of this study is to determine if in-bed cycling is safe and feasible in critically-ill patients after open heart surgery. The investigators hypothesize that in-bed cycling can ...
This pilot study investigates the use of a mobile cycling application for patients with a cardiac disease that was developed by the Expertise Centre for Digital Media of Hasselt University...
A set of opposing, nonequilibrium reactions catalyzed by different enzymes which act simultaneously, with at least one of the reactions driven by ATP hydrolysis. The results of the cycle are that ATP energy is depleted, heat is produced and no net substrate-to-product conversion is achieved. Examples of substrate cycling are cycling of gluconeogenesis and glycolysis pathways and cycling of the triglycerides and fatty acid pathways. Rates of substrate cycling may be increased many-fold in association with hypermetabolic states resulting from severe burns, cold exposure, hyperthyroidism, or acute exercise.
The introduction of new genes into cells for the purpose of treating disease by restoring or adding gene expression. Techniques include insertion of retroviral vectors, transfection, homologous recombination, and injection of new genes into the nuclei of single cell embryos. The entire gene therapy process may consist of multiple steps. The new genes may be introduced into proliferating cells in vivo (e.g., bone marrow) or in vitro (e.g., fibroblast cultures) and the modified cells transferred to the site where the gene expression is required. Gene therapy may be particularly useful for treating enzyme deficiency diseases, hemoglobinopathies, and leukemias and may also prove useful in restoring drug sensitivity, particularly for leukemia.
Preliminary cancer therapy (chemotherapy, radiation therapy, hormone/endocrine therapy, immunotherapy, hyperthermia, etc.) that precedes a necessary second modality of treatment.
Prenatal interventions to correct fetal anomalies or treat FETAL DISEASES in utero. Fetal therapies include several major areas, such as open surgery; FETOSCOPY; pharmacological therapy; INTRAUTERINE TRANSFUSION; STEM CELL TRANSPLANTATION; and GENE THERAPY.
The number of copies of a given gene present in the cell of an organism. An increase in gene dosage (by GENE DUPLICATION for example) can result in higher levels of gene product formation. GENE DOSAGE COMPENSATION mechanisms result in adjustments to the level GENE EXPRESSION when there are changes or differences in gene dosage.
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...
Biological therapy involves the use of living organisms, substances derived from living organisms, or laboratory-produced versions of such substances to treat disease. Some biological therapies for cancer use vaccines or bacteria to stimulate the body&rs...