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The quest for a therapy for muscular dystrophy has been the driving force behind the past 40 years of advances in this field. Numerous results, such as the identification of satellite cells and gene mutations that are responsible for most forms of dystrophies, advances in gene transfer and modification technology and, more recently, stem cells, have fueled hopes. However, administering corticosteroids still remains the only effective treatment available. Several recent advances have uncovered a diversity of possible therapeutic approaches, from pharmacological treatments to gene therapy (exon-skipping and adeno-associated viruses) and cell therapy with different types of newly identified stem cells. Importantly, a combination of these strategies might greatly enhance the possibility of successful therapy.  相似文献   

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Shastry BS 《IUBMB life》2000,49(6):479-484
Retinitis pigmentosa comprises a large and exceptionally heterogeneous group of hereditary disorders of the retina. As a result of an extensive investigation around the world, primary genetic lesions have been described in many genes. Some of these genes encode enzymes that are involved in the signal transduction pathway. On the basis of in vitro functional assays and standard transgenic and knock-out experiments, it has been proposed that normal cell functions are disrupted because of an abnormal protein-folding and metabolic errors or structural defects in the membrane. This ultimately leads to a gene-mediated cell death known as apoptosis. Various gene transfer approaches using mouse models further suggest that the degeneration can be rescued to some extent. Although many questions remain to be answered, investigations during the last 10 years have enormously increased our understanding of this exceptionally heterogeneous disorder and give hope for an effective gene therapy and a possible cure.  相似文献   

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Growing evidence demonstrates that psychological risk variables can contribute to physical disease. In an effort to thoroughly investigate potential etiological origins and optimal interventions, this broad review is divided into five sections: the stress response, chronic diseases, mind-body theoretical models, psychophysiological interventions, and integrated health care solutions. The stress response and its correlation to chronic disorders such as cardiovascular, gastrointestinal, autoimmune, metabolic syndrome, and chronic pain are comprehensively explored. Current mind-body theoretical models, including peripheral nerve pathway, neurophysiological, and integrative theories, are reviewed to elucidate the biological mechanisms behind psychophysiological interventions. Specific interventions included are psychotherapy, mindfulness meditation, yoga, and psychopharmacology. Finally, the author advocates for an integrated care approach as a means by which to blur the sharp distinction between physical and psychological health. Integrated care approaches can utilize psychiatric nurse practitioners for behavioral assessment, intervention, research, advocacy, consultation, and education to optimize health outcomes.  相似文献   

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W. Barr Murray 《CMAJ》1932,26(3):360-361
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Highlights► Highly variable and complex pathogens remain challenging vaccine targets. ► Systems biology and reverse vaccinology continue to foster success. ► New ways of presenting conserved, functionally critical epitopes hold promise. ► Adjuvant systems allow for enduring, native antigen presentation. ► An ideal vaccine provides synergy between innate, cellular, and humoral immunity.  相似文献   

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As a young trainee in the field of restoration ecology in the modern age, it is difficult to feel optimistic about our future. As many environmental protections are de‐regulated and the climate crisis heightens, I turned to restoration to find hope in a changing world. Restoration ecologists are the optimists of biology. We work every day to make the world a better place and our passion and forward thinking spurred the United Nation's “decade of restoration.” Learning about the successes of the hardworking members of this field gives me hope. As the earth moves toward an unimaginable future, we should continue to try to make the world a better place and encourage those around us to act and restore the environments they value, whether it be large‐scale restoration or preserving garden pollinator habitat. I am forever thankful to restoration ecology and the optimism the field provides.  相似文献   

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We propose operational definitions of reproductive optimism and pessimism; optimism involves behaving in a way that gives too much weight (in terms of producing surviving offspring) to positive events, pessimism gives too much weight to negative events. Natural selection maximizes the long-term growth of a lineage rather than short-term measures such as numbers of offspring. Consequently, optimism or pessimism can be favoured by natural selection, even though such biases appear irrational from a short-term perspective. We investigate the evolution of optimism in a metapopulation. The circumstances of a patch change over time, independently of other patches. With sufficient dispersal between patches, stochasticity affects members of a lineage largely independently and optimism is favoured. With little dispersal, the temporal fluctuations of a patch affect many members similarly; pessimism is then favoured. Our results establish that the spatial and temporal structure of the environment is crucial in determining the direction of evolved biases.  相似文献   

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