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Application and Mechanism of Action of Antisense Oligonucleotides in Precise Medicine

Zhou, H; (2020) Application and Mechanism of Action of Antisense Oligonucleotides in Precise Medicine. Journal of Precision Medicine , 35 (4) pp. 283-286. 10.13362/j.jpmed.202004001. Green open access

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Abstract

Antisense oligonucleotides are a new class of gene-targeted drugs. With the development of genomics and next-generation gene sequencing technology, as well as the emergence of various new nucleotide synthesis technologies, the application of antisense oligonucleotide drugs in translational medicine and precision medicine has achieved rapid development. Such drugs provide brand-new treatment strategies for many diseases that could not be treated in the past. Taking several representative drugs currently on the market as examples, this article focuses on explaining the main application progress and mechanism of antisense oligonucleotide drugs in the treatment of related diseases, and at the same time analyzes the problems that need to be resolved in the development of such drugs.

Type: Article
Title: Application and Mechanism of Action of Antisense Oligonucleotides in Precise Medicine
Open access status: An open access version is available from UCL Discovery
DOI: 10.13362/j.jpmed.202004001
Publisher version: https://doi.org/10.13362/j.jpmed.202004001
Language: English
Additional information: This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Oligonucleotides, antisense; Hereditary diseases, congenital; Gene therapy; Target gene repair; Molecular targeted therapy; Precision medicine;
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10112327
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