[Genetic therapy: reality and hopes].
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In recent years, five different human gene therapy clinical studies have been initiated in the United States, covering a broad spectrum of gene transfer technologies. Both in vivo and ex vivo studies have been performed, and a variety of target organs/tissues have been studied. The results of this early human clinical research indicate that there is still much to be done before a safe and effective gene therapy procedure becomes commercially available, but there is strong evidence that the obstacles that remain will not prove insurmountable. Small increases in circulating clotting factor levels have been achieved in some patients without significant side-effects, providing proof of the principle that gene therapy can provide a therapeutic benefit for patients with haemophilia. Still unclear is whether gene therapy, when it becomes available, will be accessible to the global haemophilia community. As seen with recombinant and other high purity factor concentrates, technological advances do not always lead to improvements in care for the majority of the world's haemophilia patients. In fact, advances in technology can potentially increase the gulf in care if newer processes displace existing manufacturing technologies. A modified vaccine production model should be considered to make gene therapy more widely available to those who presently have inadequate access to treatment.
BACKGROUND: Few studies have been conducted in actual clinical practice settings to evaluate the ways in which dyslipidemia is managed using lipid-modifying therapies. OBJECTIVE: To determine lipid-modifying therapy practices and their effects on low-density lipoprotein cholesterol (LDL-C) and/or total cholesterol (TC) goal attainment in Europeans based on prevailing guidelines at the time of therapy in each country. METHODS: Retrospective cohort analysis involving 58,223 patients initiated on lipid-modifying therapies in 10 European countries, with a median patient follow-up on lipid-modifying therapy of 15.3 months. Data on prescriptions of lipid-modifying therapies, laboratory data including LDL-C and TC, achievement of cholesterol goals for LDL-C and/or TC, and hospitalizations were obtained from healthcare administrative databases and/or patient chart reviews. RESULTS: Across Europe, statin monotherapy was the initial lipid-modifying treatment in 51,786 (89.3%) of 58,009 patients with available data. In addition, 38,853 (89.5%) of 43,410 patients with available follow-up statin potency data were initiated on statin regimens of medium or lower equipotency. Low-equipotency regimens include atorvastatin 5 mg, simvastatin 10 mg, and pravastatin 20 mg, whereas medium-equipotency regimens include atorvastatin 10 mg, simvastatin 20 mg, and pravastatin 40 mg. Regimens were adjusted to higher equipotency via either up-titration or switches to combination regimens in 16.2% of patients. On average, 40.5% of patients across Europe who were not initially at guideline recommended cholesterol goals (either LDL-C or TC) and had follow-up data attained recommended cholesterol levels, including <30% of patients in Spain, Italy, or Hungary. In many countries, the likelihood of goal attainment was inversely associated with baseline cardiovascular risk and/or LDL-C levels. CONCLUSIONS: Lipid management strategies in Europe during the study period were dominated by statin monotherapy. Even after prolonged follow-up on lipid-modifying therapy, approximately 60% of Europeans studied did not achieve guideline recommended cholesterol goals. Future emphasis must be placed on subsequent lipid panel monitoring, as well as the use of more efficacious, well-tolerated lipid-modifying therapies such as dual cholesterol inhibitors to enable more European patients to attain their recommended cholesterol goals.
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The goals of this study were twofold: (1) to investigate nonphobics' and phobics' physiological response in virtual environments, and (2) to analyze the trend of phobics' physiology during virtual reality (VR) treatment. As a measure of physiology, heart rate, skin resistance, and skin temperature were acquired. The data for two groups of participants were analyzed: 22 nonphobic participants (mean = 32 +/- 9.4 years) and 36 subjects with fear of flying (mean = 40 +/- 12.1 years) who met the DSM-IV criteria for fear of flying. As a result, skin resistance showed significant differences between nonphobics and phobics, T(56) = 2.978 and p < 0.01, respectively. The physiological response of 33 phobic participants, who were able to fly without medicine after VR treatment, showed a gradual trend toward the nonphobics' physiological responses as therapy sessions progressed. In this study, physiological monitoring, in particular skin resistance, appeared to be useful both in understanding the physiological state of phobic individuals and in evaluating the results of treatment in VR psychotherapy.
Chemical and pharmaceutical research have provided physicians with an array of drugs that have beneficial effects on a variety of diseases. Such drugs, however, mostly help in controlling the manifestations of the pathological condition but do not permanently modify the underlying cause. Hence the necessity of new forms of therapy that change drastically the current approach to medical treatment. Gene therapy, with its potential to correct the malfunctioning genes at the origin of variety of diseases, seems to fulfill the requirements of this therapeutic "revolution". The feasibility of such an approach is underscored by the improved knowledge of the molecular mechanism and/or gene defects at the origin of acquired diseases widely spread in the population, and of more rare congenital conditions. The technical advances in molecular biology and genetic engineering achieved in the last ten years, offer the tools necessary to implement such therapeutic interventions. Here we present the approaches currently employed for gene therapy in the context of recent clinical trials. The scientific, ethical and economical implications deriving from a prospective routine use of gene therapy in the clinical setting are discussed.
Multiple sclerosis and spinal cord injury patients can benefit by interaction with a haptic-visual system to increase the accuracy of movements in cases of spasticity, cerebellar tremor, and weakness. The device would apply a counterforce to constrain the upper extremity to a force corridor, a region of force/velocity space, designed to increase movement accuracy. Execution of movements with counterforce assistance under certain conditions improves accuracy and should enable patients to develop enhanced strategies for dealing with the movement disorders resulting from their neurologic deficits. Generation of appropriate force feedback requires dynamic adjustment of feedback plant characteristics and integration of visuospatial information in a virtual reality environment. Sensory augmentation, including compensation for visual and proprioceptive loss, can theoretically also be achieved with this approach. The underlying principles in the development of such a system are presented.
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Social workers increasingly are called on to work with mentally ill people, but in most cases they are not trained to do so. There is a significant lag between what researchers have found, particularly in the biological fields, and what educators teach in social work schools. This article describes curriculum deficits in teaching about mental illness and the devastating consequences of those deficits. It then outlines a suggested curriculum and appropriate teaching methods. Social workers are called to take the lead in curriculum reform; when they do, students will follow.
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Explore the source record for details and available documents.
Explore the source record for details and available documents.
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Virtual reality exposure therapy is slowly becoming a viable option for therapists. For virtual reality systems to be used in the daily practice of therapists, their usability needs to be taken into consideration. This paper describes the current state-of-the-art in interfaces for these systems, and describes several proposals for improving the design of these systems. An exploratory evaluation is performed to assess the merits of aspects of the proposed user interface.