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Amplifying gene expression with RNA-targeted therapeutics
Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …
from increased expression of the corresponding protein. However, in drug development, it …
Development of nucleic acid medicines based on chemical technology
Y Kawamoto, Y Wu, Y Takahashi, Y Takakura - Advanced Drug Delivery …, 2023 - Elsevier
Oligonucleotide-based therapeutics have attracted attention as an emerging modality that
includes the modulation of genes and their binding proteins related to diseases, allowing us …
includes the modulation of genes and their binding proteins related to diseases, allowing us …
Phosphorothioated DNA engineered liposomes as a general platform for stimuli‐responsive cell‐specific intracellular delivery and genome editing
A Yan, X Chen, J He, Y Ge, Q Liu… - Angewandte Chemie …, 2023 - Wiley Online Library
Intracellular protein delivery is highly desirable for protein drug‐based cell therapy.
Established technologies suffer from poor cell‐specific cytosolic protein delivery, which …
Established technologies suffer from poor cell‐specific cytosolic protein delivery, which …
Drug discovery perspectives of antisense oligonucleotides
Y Kim - Biomolecules & therapeutics, 2023 - pmc.ncbi.nlm.nih.gov
The era of innovative RNA therapies using antisense oligonucleotides (ASOs), siRNAs, and
mRNAs is beginning. Since the emergence of the concept of ASOs in 1978, it took more than …
mRNAs is beginning. Since the emergence of the concept of ASOs in 1978, it took more than …
[HTML][HTML] therapeutic oligonucleotides: An outlook on chemical strategies to improve endosomal trafficking
P Mangla, Q Vicentini, A Biscans - Cells, 2023 - mdpi.com
The potential of oligonucleotide therapeutics is undeniable as more than 15 drugs have
been approved to treat various diseases in the liver, central nervous system (CNS), and …
been approved to treat various diseases in the liver, central nervous system (CNS), and …
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery
S Qassem, D Breier, GS Naidu, I Hazan-Halevy… - … Therapy-Nucleic Acids, 2024 - cell.com
Locked nucleic acids (LNAs) are a subtype of antisense oligonucleotides (ASOs) that are
characterized by a bridge within the sugar moiety. LNAs owe their robustness to this …
characterized by a bridge within the sugar moiety. LNAs owe their robustness to this …
[HTML][HTML] Oligonucleotide-Based Therapeutics for STAT3 Targeting in Cancer—Drug Carriers Matter
S Molenda, A Sikorska, A Florczak, P Lorenc… - Cancers, 2023 - mdpi.com
Simple Summary Activated STAT3 is an essential factor in cancer development; therefore,
blocking STAT3 may be of therapeutic benefit in multiple cancer types. Thanks to recent …
blocking STAT3 may be of therapeutic benefit in multiple cancer types. Thanks to recent …
Synthesis and properties of oligonucleotides containing LNA-sulfamate and sulfamide backbone linkages
Oligonucleotides hold great promise as therapeutic agents but poor bioavailability limits
their utility. Hence, new analogues with improved cell uptake are urgently needed. Here, we …
their utility. Hence, new analogues with improved cell uptake are urgently needed. Here, we …
Synthesis, Biophysical and Biological Evaluation of Splice-Switching Oligonucleotides with Multiple LNA-Phosphothiotriester Backbones
Polyanionic antisense oligonucleotides hold great promise as RNA targeting drugs but
issues with bioavailability hinder their development. Uncharged phosphorus-based …
issues with bioavailability hinder their development. Uncharged phosphorus-based …
Non-coding RNA-directed therapeutics in lung cancer: delivery technologies and clinical applications
Lung cancer is one of the most aggressive and deadliest health threats. There has been an
increasing interest in non-coding RNA (ncRNA) recently, especially in the areas of …
increasing interest in non-coding RNA (ncRNA) recently, especially in the areas of …