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Genetic Therapies (genetic + therapy)
Selected AbstractsDuchenne's muscular dystrophy: animal models used to investigate pathogenesis and develop therapeutic strategiesINTERNATIONAL JOURNAL OF EXPERIMENTAL PATHOLOGY, Issue 4 2003C.A. Collins Summary., Duchenne's muscular dystrophy (DMD) is a lethal childhood disease caused by mutations of the dystrophin gene, the protein product of which, dystrophin, has a vital role in maintaining muscle structure and function. Homologues of DMD have been identified in several animals including dogs, cats, mice, fish and invertebrates. The most notable of these are the extensively studied mdx mouse, a genetic and biochemical model of the human disease, and the muscular dystrophic Golden Retriever dog, which is the nearest pathological counterpart of DMD. These models have been used to explore potential therapeutic approaches along a number of avenues including gene replacement and cell transplantation strategies. High-throughput screening of pharmacological and genetic therapies could potentially be carried out in recently available smaller models such as zebrafish and Caenorhabditis elegans. It is possible that a successful treatment will eventually be identified through the integration of studies in multiple species differentially suited to addressing particular questions. [source] Controlled and localized genetic manipulation in the brainJOURNAL OF CELLULAR AND MOLECULAR MEDICINE, Issue 2 2006Rachel Aronoff Abstract Brain structure and function are determined in part through experience and in part through our inherited genes. A powerful approach for unravelling the balance between activity-dependent neuronal plasticity and genetic programs is to directly manipulate the genome. Such molecular genetic studies have been greatly aided by the remarkable progress of large-scale genome sequencing efforts. Sophisticated mouse genetic manipulations allow targeted point-mutations, deletions and additions to the mouse genome. These can be regulated through inducible promoters expressing in genetically specified neuronal cell types. However, despite significant progress it remains difficult to target specific brain regions through transgenesis alone. Recent work suggests that transduction vectors, like lentiviruses and adeno-associated viruses, may provide suitable additional tools for localized and controlled genetic manipulation. Furthermore, studies with such vectors may aid the development of human genetic therapies for brain diseases. [source] Airway gene therapy and cystic fibrosisJOURNAL OF PAEDIATRICS AND CHILD HEALTH, Issue 3 2005DW Parsons Abstract:, Airway disease in cystic fibrosis (CF) is the major cause of death and is presently inadequately treatable, but genetic therapies offer the hope that such life-long disease will be curable, or at least satisfactorily treated. Normal pathogen defences that have evolved on airway surfaces also prevent the various gene vectors now available from producing effective gene transfer. Nevertheless, findings from basic research and human clinical trials are revealing how these barriers might be overcome or circumvented, with benefits to therapeutic efficacy and patient safety. Though progress is slower than expected or desired, the therapeutic rewards will be great when safe and effective gene therapy for CF airway disease becomes a clinical reality. [source] Religion, spirituality, and genetics: Mapping the terrain for research purposes,AMERICAN JOURNAL OF MEDICAL GENETICS, Issue 1 2009Larry R. Churchill Abstract Genetic diseases often raise issues of profound importance for human self-understanding, such as one's identity, the family or community to which one belongs, and one's future or destiny. These deeper questions have commonly been seen as the purview of religion and spirituality. This essay explores how religion and spirituality are understood in the current US context and defined in the scholarly literature over the past 100 years. It is argued that a pragmatic, functional approach to religion and spirituality is important to understanding how patients respond to genetic diagnoses and participate in genetic therapies. A pragmatic, functional approach requires broadening the inquiry to include anything that provides a framework of transcendent meaning for the fundamental existential questions of human life. This approach also entails suspending questions about the truth claims of any particular religious/spiritual belief or practice. Three implications of adopting this broad working definition will be presented. © 2009 Wiley-Liss, Inc. [source] An Embryonic Nation: Life Against Health in Canadian Biotechnological DiscourseCOMMUNICATION THEORY, Issue 1 2005Rebecca Sullivan This article traces the protracted public debate over reproductive and genetic technologies in Canada through an examination of the federal government's efforts to pass legislation in the area. Four attempts were made, in 1997, 2000, 2003, and finally 2004, before a bill was passed that regulated the use of embryos in both infertility treatments and nonreproductive genetic therapies. At stake in the debate was the supremacy of health over life as a fundamental value of Canadian national identity, and the role of biotechnology in ushering Canada into a new era of prosperity and global leadership. Using a feminist cultural framework, the author challenges notions of modernity versus postmodernity in the social construction of bodies, nations, and knowledge. She critiques the legal intrusions on women's bodies in particular for the way that they, perhaps inadvertently, offer some limited form of autonomy for embryos as valuable commodities in scientific progress. [source] Gene therapy for diseases of the cornea , a reviewCLINICAL & EXPERIMENTAL OPHTHALMOLOGY, Issue 2 2010Keryn A Williams PhD Abstract The cornea is particularly suited to gene therapy. The cornea is readily accessible, normally transparent, and is somewhat sequestrated from the general circulation and the systemic immune system. The principle of genetic therapy for the cornea is to use an appropriate vector system to transfer a gene to the cornea itself, or to the ocular environs, or systemically, so that a transgenic protein will be expressed that will modulate congenital or acquired disease. The protein may be structural such as a collagen, or functionally active such as an enzyme, cytokine or growth factor that may modulate a pathological process. Alternatively, gene expression may be silenced by the use of modalities such as antisense oligonucleotides. Interestingly, despite a very considerable amount of work in animal models, clinical translation directed to gene therapy of the human cornea has been minimal. This is in contrast to gene therapy for monogenic inherited diseases of the retina, where promising early results of clinical trials for Leber's congenital amaurosis have already been published and a number of other trials are ongoing. [source] | ||||||||||