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Peptide-DNA Co-Assembled Nanoparticles as a Nonviral Vector for Gene Delivery with High Transgene Expression
ACS Applied Nano Materials ( IF 5.3 ) Pub Date : 2023-02-17 , DOI: 10.1021/acsanm.2c04279
Rong Ni 1 , Ruilu Feng 1 , Ying Chau 1
ACS Applied Nano Materials ( IF 5.3 ) Pub Date : 2023-02-17 , DOI: 10.1021/acsanm.2c04279
Rong Ni 1 , Ruilu Feng 1 , Ying Chau 1
Affiliation
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Nonviral vectors for gene therapy are a safer alternative to viral vectors that elicit a harmful immune response. Artificial viruses combine the strengths of viral and nonviral vectors and have emerged to be safe and efficient gene delivery vectors. Herein, we report the development of peptide–DNA coassembled nanoparticles as a new efficient vector for gene delivery with high transgene efficiency. The key to our success is to introduce several rationally designed functional short peptide modules: (1) a pH-sensitive segment, (2) an endosomal interruption segment, (3) a cell penetration peptide with high positive charges, (4) a nuclear localization signal peptide with positive charges, (5) aromatic groups to tune the hydrophobic/hydrophilic ratio, and (6) redox-sensitive residues. These peptide–DNA coassembled nanoparticles achieved efficient DNA delivery with transfection efficiency over 90% in a serum-containing medium. This work demonstrates the effectiveness of the module-based design approach and lays the groundwork for potential biomedical applications of peptide-based biomaterials.
中文翻译:
肽-DNA 共组装纳米颗粒作为非病毒载体,用于高转基因表达的基因传递
用于基因治疗的非病毒载体是引发有害免疫反应的病毒载体的更安全替代品。人工病毒结合了病毒和非病毒载体的优势,已成为安全高效的基因传递载体。在此,我们报告了肽-DNA 共组装纳米粒子作为一种新的高效基因传递载体的开发,具有高转基因效率。我们成功的关键在于引入了几个合理设计的功能性短肽模块:(1) pH 敏感片段,(2) 内体中断片段,(3) 具有高正电荷的细胞穿透肽,(4) 核带正电荷的定位信号肽,(5) 用于调节疏水/亲水比的芳香族基团,以及 (6) 氧化还原敏感残基。这些肽-DNA 共组装纳米颗粒实现了高效的 DNA 递送,在含血清的培养基中转染效率超过 90%。这项工作证明了基于模块的设计方法的有效性,并为基于肽的生物材料的潜在生物医学应用奠定了基础。
更新日期:2023-02-17
中文翻译:
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肽-DNA 共组装纳米颗粒作为非病毒载体,用于高转基因表达的基因传递
用于基因治疗的非病毒载体是引发有害免疫反应的病毒载体的更安全替代品。人工病毒结合了病毒和非病毒载体的优势,已成为安全高效的基因传递载体。在此,我们报告了肽-DNA 共组装纳米粒子作为一种新的高效基因传递载体的开发,具有高转基因效率。我们成功的关键在于引入了几个合理设计的功能性短肽模块:(1) pH 敏感片段,(2) 内体中断片段,(3) 具有高正电荷的细胞穿透肽,(4) 核带正电荷的定位信号肽,(5) 用于调节疏水/亲水比的芳香族基团,以及 (6) 氧化还原敏感残基。这些肽-DNA 共组装纳米颗粒实现了高效的 DNA 递送,在含血清的培养基中转染效率超过 90%。这项工作证明了基于模块的设计方法的有效性,并为基于肽的生物材料的潜在生物医学应用奠定了基础。