A new developing class of gene delivery: messenger RNA-based therapeutics,Biomaterials Science

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A new developing class of gene delivery: messenger RNA-based therapeutics
Biomaterials Science ( IF 5.8 ) Pub Date : 2017-10-02 00:00:00 , DOI: 10.1039/c7bm00712d
Zhao Meng _{1,

2,

3,

4,

5} , Jonathan O'Keeffe-Ahern _{5,

6,

7,

8,

9} , Jing Lyu _{1,

2,

3,

4,

5} , Luca Pierucci _{5,

6,

7,

8,

9} , Dezhong Zhou _{5,

6,

7,

8,

9} , Wenxin Wang _{1,

2,

3,

4,

5}

Affiliation

Gene therapy has long been held as having the potential to become a front line treatment for various genetic disorders. However, the direct delivery of nucleic acids to correct a genetic disorder has numerous limitations owing to the inability of naked nucleic acids (DNA and RNA) to traverse the cell membrane. Recently, messenger RNA (mRNA) based delivery has become a more attractive alternative to DNA due to the relatively easier transfection process, higher efficiency and safety profile. As with all gene therapies, the central challenge that remains is the efficient delivery of nucleic acids intracellularly. This review presents the recent progress in mRNA delivery, focusing on comparing the advantages and limitations of non-viral based delivery vectors.

中文翻译：

基因递送的新发展类别：基于信使RNA的治疗剂

长期以来，基因治疗被认为具有成为各种遗传疾病的一线治疗的潜力。然而，由于裸核酸（DNA和RNA）不能穿过细胞膜，因此直接递送核酸以纠正遗传疾病具有许多限制。最近，由于相对较容易的转染过程，更高的效率和安全性，基于信使RNA（mRNA）的递送已成为DNA的更有吸引力的替代方案。与所有基因疗法一样，仍然面临的主要挑战是细胞内核酸的有效递送。这篇综述介绍了mRNA传递的最新进展，重点是比较基于非病毒的传递载体的优点和局限性。

更新日期：2017-10-24

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