TY - JOUR
T1 - Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology
AU - Weng, Yuhua
AU - Huang, Qianqian
AU - Li, Chunhui
AU - Yang, Yongfeng
AU - Wang, Xiaoxia
AU - Yu, Jie
AU - Huang, Yuanyu
AU - Liang, Xing Jie
N1 - Publisher Copyright:
© 2019 The Author(s)
PY - 2020/3/6
Y1 - 2020/3/6
N2 - Due to a series of systemic and intracellular obstacles in nucleic acid (NA) therapy, including fast degradation in blood, renal clearance, poor cellular uptake, and inefficient endosomal escape, NAs may need delivery methods to transport to the cell nucleus or cytosol to be effective. Advanced nanoscale biotechnology-associated strategies, such as controlling the particle size, charge, drug loading, response to environmental signals, or other physical/chemical properties of delivery carriers, have provided great help for the in vivo and in vitro delivery of NA therapeutics. In this review, we introduce the characteristics of different NA modalities and illustrate how advanced nanoscale biotechnology assists NA therapy. The specific features and challenges of various nanocarriers in clinical and preclinical studies are summarized and discussed. With the help of advanced nanoscale biotechnology, some of the major barriers to the development of NA therapy will eventually be overcome in the near future.
AB - Due to a series of systemic and intracellular obstacles in nucleic acid (NA) therapy, including fast degradation in blood, renal clearance, poor cellular uptake, and inefficient endosomal escape, NAs may need delivery methods to transport to the cell nucleus or cytosol to be effective. Advanced nanoscale biotechnology-associated strategies, such as controlling the particle size, charge, drug loading, response to environmental signals, or other physical/chemical properties of delivery carriers, have provided great help for the in vivo and in vitro delivery of NA therapeutics. In this review, we introduce the characteristics of different NA modalities and illustrate how advanced nanoscale biotechnology assists NA therapy. The specific features and challenges of various nanocarriers in clinical and preclinical studies are summarized and discussed. With the help of advanced nanoscale biotechnology, some of the major barriers to the development of NA therapy will eventually be overcome in the near future.
KW - ASO
KW - CRISPR/Cas
KW - RNAi
KW - drug delivery
KW - gene editing
KW - nucleic acid therapy
UR - https://www.scopus.com/pages/publications/85077650872
U2 - 10.1016/j.omtn.2019.12.004
DO - 10.1016/j.omtn.2019.12.004
M3 - Review article
AN - SCOPUS:85077650872
SN - 2162-2531
VL - 19
SP - 581
EP - 601
JO - Molecular Therapy Nucleic Acids
JF - Molecular Therapy Nucleic Acids
ER -