Adenoviral Vectors for Gene Therapy of Hereditary Diseases

被引:0
|
作者
Muravyeva, Anna [1 ]
Smirnikhina, Svetlana [1 ]
机构
[1] Res Ctr Med Genet, Lab Genome Editing, Moskvorechye 1, Moscow 115522, Russia
来源
BIOLOGY-BASEL | 2024年 / 13卷 / 12期
关键词
adenoviral vector; gene therapy; hereditary diseases; cystic fibrosis; ornithine transcarbamylase deficiency; TRANSMEMBRANE CONDUCTANCE REGULATOR; HUMAN CYSTIC-FIBROSIS; NONHUMAN-PRIMATES; APICAL SURFACE; EXPRESSION; RECEPTOR; CELL; DELIVERY; LUNG; PROTEINS;
D O I
10.3390/biology13121052
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found their place in gene therapy for hereditary diseases. This review provides an overview of AdVs, their features, and clinical trials using them for gene replacement therapy in monogenic diseases and analyzes the reasons for the failures of these studies. Additionally, current research on the modification of AdVs to reduce immune responses and target delivery is discussed.
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页数:19
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