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用于治疗囊性纤维化的吸入性生物制剂。

Inhaled Biologicals for the Treatment of Cystic Fibrosis.

作者信息

Sala Valentina, Murabito Alessandra, Ghigo Alessandra

机构信息

Department of Molecular Biotechnology and Health Sciences, Molecular Biotechnology Center, University of Torino, Torino, Italy.

S.C. Medicina d'Urgenza, A.O.U. Città della Salute e della Scienza, Molinette Hospital, Torino, Italy.

出版信息

Recent Pat Inflamm Allergy Drug Discov. 2019;13(1):19-26. doi: 10.2174/1872213X12666181012101444.

DOI:10.2174/1872213X12666181012101444
PMID:30318010
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6751348/
Abstract

BACKGROUND

Cystic Fibrosis (CF), one of the most frequent genetic diseases, is characterized by the production of viscous mucus in several organs. In the lungs, mucus clogs the airways and traps bacteria, leading to recurrent/resistant infections and lung damage. For cystic fibrosis patients, respiratory failure is still lethal in early adulthood since available treatments display incomplete efficacy.

OBJECTIVE

The objective of this review is to extend the current knowledge in the field of available treatments for cystic fibrosis. A special focus has been given to inhaled peptide-based drugs.

METHODS

The current review is based on recent and/or relevant literature and patents already available in various scientific databases, which include PubMed, PubMed Central, Patentscope and Science Direct. The information obtained through these diverse databases is compiled, critically interpreted and presented in the current study. An in-depth but not systematic approach to the specific research question has been adopted.

RESULTS

Recently, peptides have been proposed as possible pharmacologic agents for the treatment of respiratory diseases. Of note, peptides are suitable to be administered by inhalation to maximize efficacy and reduce systemic side effects. Moreover, innovative delivery carriers have been developed for drug administration through inhalation, allowing not only protection against proteolysis, but also a prolonged and controlled release.

CONCLUSION

Here, we summarize newly patented peptides that have been developed in the last few years and advanced technologies for inhaled drug delivery to treat cystic fibrosis.

摘要

背景

囊性纤维化(CF)是最常见的遗传性疾病之一,其特征是多个器官产生黏稠的黏液。在肺部,黏液会堵塞气道并困住细菌,导致反复/耐药感染和肺部损伤。对于囊性纤维化患者来说,呼吸衰竭在成年早期仍然是致命的,因为现有的治疗方法疗效并不完全。

目的

本综述的目的是扩展目前在囊性纤维化可用治疗领域的知识。特别关注了基于肽的吸入药物。

方法

本综述基于各种科学数据库中已有的近期和/或相关文献及专利,这些数据库包括PubMed、PubMed Central、Patentscope和ScienceDirect。通过这些不同数据库获得的信息在本研究中进行了整理、批判性解读和呈现。对具体研究问题采用了深入但非系统的方法。

结果

最近,肽已被提议作为治疗呼吸系统疾病的可能药物。值得注意的是,肽适合通过吸入给药,以最大化疗效并减少全身副作用。此外,已经开发出用于吸入给药的创新递送载体,不仅可以防止蛋白水解,还能实现延长和控释。

结论

在此,我们总结了过去几年开发的新获专利的肽以及用于吸入给药治疗囊性纤维化的先进技术。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2a00/6751348/f03a3c7873eb/PRIAD-13-19_F1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2a00/6751348/f03a3c7873eb/PRIAD-13-19_F1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2a00/6751348/f03a3c7873eb/PRIAD-13-19_F1.jpg

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