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Stereopure AIMer: A Promising RNA Base-editing Tool for Monogenic Neurological Diseases

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References

  1. Monian P, Shivalila C, Lu G, Shimizu M, Boulay D, Bussow K, et al. Endogenous ADAR-mediated RNA editing in non-human Primates using stereopure chemically modified oligonucleotides. Nat Biotechnol 2022: 1–10.

  2. Iwamoto N, Butler DCD, Svrzikapa N, Mohapatra S, Zlatev I, Sah DWY. Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides. Nat Biotechnol 2017, 35: 845–851.

    Article  CAS  Google Scholar 

  3. Qu L, Yi Z, Zhu S, Wang C, Cao Z, Zhou Z, et al. Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs. Nat Biotechnol 2019, 37: 1059–1069.

    Article  CAS  Google Scholar 

  4. Merkle T, Merz S, Reautschnig P, Blaha A, Li Q, Vogel P, et al. Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides. Nat Biotechnol 2019, 37: 133–138.

    Article  CAS  Google Scholar 

  5. Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: An overview and prospectus. Nat Rev Drug Discov 2021, 20: 427–453.

    Article  CAS  Google Scholar 

  6. Bennett CF, Krainer AR, Cleveland DW. Antisense oligonucleotide therapies for neurodegenerative diseases. Annu Rev Neurosci 2019, 42: 385–406.

    Article  CAS  Google Scholar 

  7. Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, DeNizio JE, Reiss CW, et al. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in Primates. Nature 2021, 593: 429–434.

    Article  CAS  Google Scholar 

  8. Qin Y, Du Y, Chen L, Liu Y, Xu W, Liu Y, et al. A recurrent SHANK1 mutation implicated in autism spectrum disorder causes autistic-like core behaviors in mice via downregulation of mGluR1-IP3R1-calcium signaling. Mol Psychiatry 2022, 27: 2985–2998.

    Article  CAS  Google Scholar 

  9. Melchionda S, Ahituv N, Bisceglia L, Sobe T, Glaser F, Rabionet R, et al. MYO6, the human homologue of the gene responsible for deafness in Snell’s waltzer mice, is mutated in autosomal dominant nonsyndromic hearing loss. Am J Hum Genet 2001, 69: 635–640.

    Article  CAS  Google Scholar 

  10. Wang C, Li Y, Shi L, Ren J, Patti M, Wang T, et al. Mutations in SLC20A2 link familial idiopathic basal Ganglia calcification with phosphate homeostasis. Nat Genet 2012, 44: 254–256.

    Article  CAS  Google Scholar 

  11. Kuijper EC, Bergsma AJ, Pijnappel WWMP, Aartsma-Rus A. Opportunities and challenges for antisense oligonucleotide therapies. J Inherit Metab Dis 2021, 44: 72–87.

    Article  CAS  Google Scholar 

  12. Min HS, Kim HJ, Naito M, Ogura S, Toh K, Hayashi K, et al. Systemic brain delivery of antisense oligonucleotides across the blood-brain barrier with a glucose-coated polymeric nanocarrier. Angew Chem Int Ed Engl 2020, 59: 8173–8180.

    Article  CAS  Google Scholar 

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Acknowledgements

We thank Yong Chen from Rib Lab for cooperation, discussion, and schematic drawing during this investigation. This work was supported by grants from the Ministry of Science and Technology of China (2018YFA0107900, 92168103, 32171417, 82001140 and 2019CXJQ01), the National Nature Science Foundation, and Shanghai Municipal Government, Peak Disciplines (Type IV) of Institutions of Higher Leaning in Shanghai.

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Correspondence to Jianhong Zhu.

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Yu, J., Li, T., Chen, K. et al. Stereopure AIMer: A Promising RNA Base-editing Tool for Monogenic Neurological Diseases. Neurosci. Bull. 39, 353–355 (2023). https://doi.org/10.1007/s12264-022-00935-5

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  • DOI: https://doi.org/10.1007/s12264-022-00935-5

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