囊性纤维化
遗传增强
基因传递
基因组编辑
计算生物学
医学
基因
生物信息学
生物
基因组
遗传学
作者
Namratha Turuvekere Vittala Murthy,Kseniya Yu. Vlasova,Jordan B. Renner,Antony Jozić,Gaurav Sahay
标识
DOI:10.1016/j.addr.2024.115305
摘要
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.
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