Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9905518 | PMC |
| http://dx.doi.org/10.1007/s12264-022-00935-5 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Stereopure AIMer: A Promising RNA Base-editing Tool for Monogenic Neurological Diseases.
Neurosci Bull
February 2023
Department of Neurosurgery, Huashan Hospital, Shanghai Medical College, Fudan University, National Center for Neurological Disorders, National Key Laboratory for Medical Neurobiology, Institutes of Brain Science, Shanghai Key Laboratory of Brain Function and Regeneration, Institute of Neurosurgery, MOE Frontiers Center for Brain Science, Shanghai, 200000, China.
Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides.
Nat Biotechnol
July 2022
Wave Life Sciences, Cambridge, MA, USA.
Technologies that recruit and direct the activity of endogenous RNA-editing enzymes to specific cellular RNAs have therapeutic potential, but translating them from cell culture into animal models has been challenging. Here we describe short, chemically modified oligonucleotides called AIMers that direct efficient and specific A-to-I editing of endogenous transcripts by endogenous adenosine deaminases acting on RNA (ADAR) enzymes, including the ubiquitously and constitutively expressed ADAR1 p110 isoform. We show that fully chemically modified AIMers with chimeric backbones containing stereopure phosphorothioate and nitrogen-containing linkages based on phosphoryl guanidine enhanced potency and editing efficiency 100-fold compared with those with uniformly phosphorothioate-modified backbones in vitro.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!