收藏切换
Application of base editors in the treatment of genetic disorders
收藏切换
PDF
Yuan CHEN, Yu-xuan CHEN, Yuan PING*
Acta Pharmaceutica Sinica | 2020, 55(7) : 1562 - 1572
Less
收藏切换
Acta Pharmaceutica Sinica | 2020, 55(7): 1562-1572
Reviews
Application of base editors in the treatment of genetic disorders
Full
Yuan CHEN, Yu-xuan CHEN, Yuan PING*
Affiliations
  • College of Pharmaceutical Sciences, Zhejiang University, Hangzhou 310058, China
Published: 2020-07-12 doi: 10.16438/j.0513-4870.2020-0942
Outline
收藏切换

To date, CRISPR/Cas systems represent the most widely used tool for genome editing; however, its application scope for gene therapy has been largely limited due to its limited efficiency in activating homologydirected repair for DNA and off-target effect. Base editing is a new CRISPR/Cas-based genome-editing strategy, which allows single nucleotide to be precisely corrected in a narrow window scope on the target DNA or RNA by taking advantage of different nucleobase deaminases. Base editors include cytosine base editors (CBEs) and adenine base editors (ABEs), which can induce the conversions from C·G to T·A and A·T to G·C, respectively. Base editors work independently of double-strand DNA breaks (DSBs) and DNA donor templates, and thus they are extensively adopted for a wide range of therapeutic applications for genetic diseases, largely owing to their high efficiency and great specificity. In this review, we summarize the development of base editors and their potentials as therapeutic drugs for treating genetic diseases, and future outlooks are also discussed.

CRISPR/Cas9  /  gene editing  /  gene therapy  /  base editor  /  genetic disorder
Yuan CHEN, Yu-xuan CHEN, Yuan PING. Application of base editors in the treatment of genetic disorders[J]. Acta Pharmaceutica Sinica, 2020 , 55 (7) : 1562 -1572 . DOI: 10.16438/j.0513-4870.2020-0942
Year 2020 volume 55 Issue 7
PDF
161
70
Cite this Article
BibTeX
Article Info
doi: 10.16438/j.0513-4870.2020-0942
  • Receive Date:2020-06-09
  • Online Date:2026-01-21
  • Published:2020-07-12
Article Data
Affiliations
History
  • Received:2020-06-09
  • Revised:2020-06-25
Funding
Affiliations
    College of Pharmaceutical Sciences, Zhejiang University, Hangzhou 310058, China
References
Share
https://castjournals.cast.org.cn/joweb/yxxb/EN/10.16438/j.0513-4870.2020-0942
Share to
QR

Scan QR to access full text

Cite this article
BibTeX
Citations
表12种不同金属材料的力学参数

Family
属数
Number of
genus
种数
Number of
species
占总种数比例
Percentage of
total species (%)

Genus
种数
Number of
species
占总种数比例
Percentage of total
species (%)
鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
关闭全屏
  • BibTeX
  • EndNote
  • RefWorks
  • TxT