Rodríguez-Fragoso L
Departamento de Gastroenterología, Instituto Nacional de la Nutrición Salvador Zubirán, México, D.F.
Rev Invest Clin. 1997 Sep-Oct;49(5):397-409.
In the last years there has been much progress in our understanding of molecular mechanisms in the pathogenesis of disease. In this review we provide an overview of gene therapy, its most actualized techniques for gene delivery, and we give specific examples of laboratory and clinical achievements to date. The development of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. As a result, concepts and methods that would have been considered purely science fiction 50 years ago are now used in the treatment of diseases. The widespread application of gene therapy technology to many diseases is already breaking down the traditional boundaries of modern medicine. However, despite its progress, several key technical drawbacks need to be overcome before gene therapy can be used safely and effectively in clinical settings. Technological developments, particularly in the areas of gene delivery and cell transplantation, will be critical for the successful practice of gene therapy.
在过去的几年里，我们对疾病发病机制中的分子机制的理解取得了很大进展。在这篇综述中，我们概述了基因治疗、其最先进的基因递送技术，并给出了迄今为止实验室和临床成果的具体例子。将基因递送至哺乳动物细胞的方法的发展激发了人们对通过基于基因的疗法治疗人类疾病可能性的极大兴趣。因此，50年前还会被认为是纯粹科幻小说的概念和方法现在已被用于疾病治疗。基因治疗技术在许多疾病中的广泛应用正在打破现代医学的传统界限。然而，尽管取得了进展，但在基因治疗能够在临床环境中安全有效地使用之前，仍有几个关键的技术缺陷需要克服。技术发展，特别是在基因递送和细胞移植领域的发展，对于基因治疗的成功应用至关重要。
