Oligonucleotide-directed RNA editing in primates.
Oligonucleotide-directed RNA editing in primates.
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DOI：
10.1016/j.ymthe.2022.04.005
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发表时间：
2022-06-01
影响因子：
12.4
通讯作者：
Beal, Peter A.
中科院分区：
文献类型：
--
作者：
Doherty, Erin E.;Beal, Peter A.
Recent approvals of antisense, exon skipping, and small interfering RNA (siRNA) therapeutics, along with the highly successful mRNA-based COVID-19 vaccines, have invigorated the field of nucleic acid therapeutics and stimulated interest in other mechanisms by which oligonucleotides might elicit therapeutic effects. 1 Oligonucleotides can be used to guide Adenosine Deaminases Acting on RNA (ADARs) to correct disease-causing mutations, suggesting the possibility of therapeutic RNA editing. 2–5 The manuscript by Monian and Shivalila et al. recently published in Nature Biotechnology describes the recruitment of endogenously expressed ADARs with short, chemically modified oligonucleotides (AIMers) for efficient directed RNA editing in non-human primates (NHPs) for the first time. Importantly, the authors show that a fully modified 30-nucleotide (nt) AIMer bearing GalNAc to facilitate liver uptake yielded 50% target transcript editing without measurable off-target editing. 6 This advance sets the stage for translation of oligonucleotide-directed RNA editing technologies to the clinic.The ADAR family of enzymes deaminates adenosine (A) to inosine (I) in doublestranded RNA. Inosine is recognized during translation as guanosine (G), thereby affording an A-to-G transition. This is significant because most human genetic variants associated with disease are point mutations, a majority of which could be reversed by A-to-G transitions. 7 In addition, recent reports detailing genomic editing of the pathogenic sickle cell hemoglobin allele to another natu-
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影响因子：
15
作者：
Doherty EE;Wilcox XE;van Sint Fiet L;Kemmel C;Turunen JJ;Klein B;Tantillo DJ;Fisher AJ;Beal PA
通讯作者：
Beal PA
影响因子：
46.9
作者：
Monian, Prashant;Shivalila, Chikdu;Vargeese, Chandra
通讯作者：
Vargeese, Chandra
影响因子：
48
作者：
Katrekar, Dhruva;Chen, Genghao;Mali, Prashant
通讯作者：
Mali, Prashant
影响因子：
5.7
作者：
Damase TR;Sukhovershin R;Boada C;Taraballi F;Pettigrew RI;Cooke JP
通讯作者：
Cooke JP
DOI：
10.1038/s41576-018-0059-1
发表时间：
2018-12
期刊：
Nature reviews. Genetics
影响因子：
--
作者：
Rees HA;Liu DR
通讯作者：
Liu DR