Considerations in the preclinical assessment of the safety of antisense oligonucleotides
A Goyenvalle, C Jimenez-Mallebrera… - nucleic acid …, 2023 - liebertpub.com
The nucleic acid therapeutics field has made tremendous progress in the past decades.
Continuous advances in chemistry and design have led to many successful clinical …
Continuous advances in chemistry and design have led to many successful clinical …
Stability prediction of canonical and non-canonical structures of nucleic acids in various molecular environments and cells
S Takahashi, N Sugimoto - Chemical Society Reviews, 2020 - pubs.rsc.org
Nucleic acids (DNA and RNA) dynamically fold and unfold to exert their functions in cells.
These folding and unfolding behaviours are also the basis for various technical applications …
These folding and unfolding behaviours are also the basis for various technical applications …
[HTML][HTML] Specificity of oligonucleotide gene therapy (OGT) agents
DD Nedorezova, MV Dubovichenko, EP Belyaeva… - Theranostics, 2022 - ncbi.nlm.nih.gov
Oligonucleotide gene therapy (OGT) agents (eg antisense, deoxyribozymes, siRNA and
CRISPR/Cas) are promising therapeutic tools. Despite extensive efforts, only few OGT drugs …
CRISPR/Cas) are promising therapeutic tools. Despite extensive efforts, only few OGT drugs …
[HTML][HTML] Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry
C Terada, K Oh, R Tsubaki, B Chan, N Aibara… - Nature …, 2023 - nature.com
Off-target interactions between antisense oligonucleotides (ASOs) with state-of-the-art
modifications and biological components still pose clinical safety liabilities. To mitigate a …
modifications and biological components still pose clinical safety liabilities. To mitigate a …
Transcriptome-wide off-target effects of steric-blocking oligonucleotides
Steric-blocking oligonucleotides (SBOs) are short, single-stranded nucleic acids designed to
modulate gene expression by binding to RNA transcripts and blocking access from cellular …
modulate gene expression by binding to RNA transcripts and blocking access from cellular …
Nuclear magnetic resonance spectra and AMBER OL3 and ROC-RNA simulations of UCUCGU reveal force field strengths and weaknesses for single-stranded RNA
J Zhao, SD Kennedy, DH Turner - Journal of Chemical Theory and …, 2022 - ACS Publications
Single-stranded regions of RNA are important for folding of sequences into 3D structures
and for design of therapeutics targeting RNA. Prediction of ensembles of 3D structures for …
and for design of therapeutics targeting RNA. Prediction of ensembles of 3D structures for …
Improved nearest-neighbor parameters for the stability of RNA/DNA hybrids under a physiological condition
D Banerjee, H Tateishi-Karimata… - Nucleic Acids …, 2020 - academic.oup.com
Abstract The stability of Watson–Crick paired RNA/DNA hybrids is important for designing
optimal oligonucleotides for ASO (Antisense Oligonucleotide) and CRISPR (Clustered …
optimal oligonucleotides for ASO (Antisense Oligonucleotide) and CRISPR (Clustered …
Nearest-neighbor parameters for predicting DNA duplex stability in diverse molecular crowding conditions
S Ghosh, S Takahashi, T Ohyama… - Proceedings of the …, 2020 - National Acad Sciences
The intracellular environment is crowded and heterogeneous. Although the thermodynamic
stability of nucleic acid duplexes is predictable in dilute solutions, methods of predicting …
stability of nucleic acid duplexes is predictable in dilute solutions, methods of predicting …
[PDF][PDF] Preclinical safety assessment of therapeutic oligonucleotides
P Andersson - Antisense RNA Design, Delivery, and Analysis, 2022 - library.oapen.org
During the last decade, therapeutic oligonucleotide drugs (OND) have witnessed a
tremendous development in chemistry and mechanistic understanding that have translated …
tremendous development in chemistry and mechanistic understanding that have translated …
Using RNA-seq to assess off-target effects of antisense oligonucleotides in human cell lines
S Michel, K Schirduan, Y Shen, R Klar, J Tost… - Molecular Diagnosis & …, 2021 - Springer
Background The field of antisense oligonucleotide therapeutics is rapidly growing and in
addition to small molecules and therapeutic antibodies, oligonucleotide-based gene …
addition to small molecules and therapeutic antibodies, oligonucleotide-based gene …