A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines,Advanced Drug Delivery Reviews

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A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines
Advanced Drug Delivery Reviews ( IF 15.2 ) Pub Date : 2024-04-16 , DOI: 10.1016/j.addr.2024.115305
Namratha Turuvekere Vittala Murthy ₁ , Kseniia Vlasova ₁ , Jonas Renner ₁ , Antony Jozic ₁ , Gaurav Sahay ₂

Affiliation

Cystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative gene delivery strategies to address the mutations in the gene. This review delves into the promises and challenges of non-viral gene delivery for CF therapy and explores strategies to overcome these hurdles. Several emerging technologies and nucleic acid cargos for CF gene therapy are discussed. Novel formulation approaches including lipid and polymeric nanoparticles promise enhanced delivery through the CF mucus barrier, augmenting the potential of non-viral strategies. Additionally, safety considerations and regulatory perspectives play a crucial role in navigating the path toward clinical translation of gene therapy.

中文翻译：

通过基因组药物的非病毒递送靶向囊性纤维化的新时代

囊性纤维化 (CF) 是一种复杂的遗传性呼吸系统疾病，需要创新的基因递送策略来解决基因突变。本综述深入探讨了 CF 治疗的非病毒基因递送的前景和挑战，并探讨了克服这些障碍的策略。讨论了用于 CF 基因治疗的几种新兴技术和核酸货物。包括脂质和聚合物纳米颗粒在内的新型制剂方法有望增强通过 CF 粘液屏障的递送，从而增强非病毒策略的潜力。此外，安全性考虑和监管观点在基因治疗临床转化的道路上发挥着至关重要的作用。

更新日期：2024-04-16

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