Pulmonary delivery of sirna for the treatment of cystic fibrosis and pulmonary delivery platforms

Nensi Raytthatha, Isha Shah, Jigar Vyas
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Abstract

Cystic fibrosis (CF) is one of the most deadly diseases of lungs that involves symptoms such as breathing difficulties, coughing and lung infection. Despite important therapeutic advances, the definitive treatment for CF remains elusive. CF is a good candidate for gene therapy because it is relatively common, lethal and monogenic and it does not have adequate treatment options. In this review article, we have reviewed gene therapy as a potential treatment option for CF. Various platforms and strategies for pulmonary gene delivery have also been discussed in detail.
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肺输送sirna治疗囊性纤维化及肺输送平台
囊性纤维化(CF)是最致命的肺部疾病之一,其症状包括呼吸困难、咳嗽和肺部感染。尽管有重要的治疗进展,但CF的最终治疗仍然难以捉摸。CF是一个很好的基因治疗候选者,因为它是相对常见的,致命的和单基因的,它没有足够的治疗选择。在这篇综述文章中,我们回顾了基因治疗作为CF的潜在治疗选择,并详细讨论了肺基因传递的各种平台和策略。
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