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首页> 外文期刊>Peritoneal dialysis international: Journal of the International Society for Peritoneal Dialysis >The potential of gene therapy in the peritoneal cavity.
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The potential of gene therapy in the peritoneal cavity.

机译：腹膜腔内基因治疗的潜力。

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摘要

Gene therapy is a promising new treatment modality based on molecular genetic modification to achieve a therapeutic benefit. We believe that gene therapy in the peritoneal cavity holds considerable promise, and we describe strategies by which genetic modification can be used to treat a variety of disease states or conditions. First, we can envision a strategy, based on genetic modification of the peritoneal membrane, to improve the practice of peritoneal dialysis through the production of proteins that would be of therapeutic value in preventing membrane damage and in preserving or enhancing its function as a dialyzing membrane. Second, the membrane could be genetically modified for either local or systemic delivery of therapeutic proteins. This approach could be applied to a variety of pathologies or conditions that require either sustained or transient delivery of therapeutic proteins, such as enzymes or growth factors. Third, gene transfer has already been incorporated into several strategies for the treatment of intra-abdominal carcinomas, and it has been effective in animal models of ovarian and bladder cancer and of peritoneal mesothelioma. Finally, gene transfer can be a valuable tool in increasing our understanding of the biology of the peritoneal membrane. By being able to manipulate the expression of specific genes through gene transfer, their role in various (patho)physiological processes can be identified. In summary, gene therapy in the peritoneal cavity has significant potential to address a variety of diseases or pathophysiological conditions, and to further our knowledge of peritoneal cavity biology.
机译：基因疗法是一种基于分子遗传修饰以实现治疗益处的有前途的新治疗方式。我们相信腹膜腔内的基因治疗具有广阔的前景，并且我们描述了可以通过基因修饰来治疗多种疾病或病症的策略。首先，我们可以设想一种基于腹膜遗传修饰的策略，通过产生蛋白质来改善腹膜透析的实践，该蛋白质在预防膜损伤以及保持或增强其透析膜功能方面具有治疗价值。 。第二，可以对膜进行遗传修饰以用于治疗性蛋白的局部或全身递送。这种方法可以应用于需要持续或短暂递送治疗性蛋白质（例如酶或生长因子）的各种病理或状况。第三，基因转移已被纳入治疗腹腔内癌的几种策略中，并且已在卵巢癌和膀胱癌以及腹膜间皮瘤的动物模型中有效。最后，基因转移可以成为增加我们对腹膜膜生物学理解的有价值的工具。通过能够通过基因转移操纵特定基因的表达，可以确定它们在各种（病理）生理过程中的作用。总而言之，腹膜腔内的基因治疗在解决各种疾病或病理生理状况以及进一步了解腹膜腔生物学方面具有巨大潜力。

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