Amplifying gene expression with RNA-targeted therapeutics

O Khorkova, J Stahl, A Joji, CH Volmar… - Nature Reviews Drug …, 2023 - nature.com
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 …

Non-coding RNA-directed therapeutics in lung cancer: delivery technologies and clinical applications

AAH Abdellatif, G Scagnetti, MA Younis… - Colloids and Surfaces B …, 2023 - Elsevier
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 …

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 …

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 …

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 …

[HTML][HTML] Drug discovery perspectives of antisense oligonucleotides

Y Kim - Biomolecules & Therapeutics, 2023 - 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 …

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 …

MolBiC: the cell-based landscape illustrating molecular bioactivities

Y Ge, M Yang, X Yu, Y Zhou, Y Zhang… - Nucleic Acids …, 2024 - academic.oup.com
The measurement of cell-based molecular bioactivity (CMB) is critical for almost every step
of drug development. With the booming application of AI in biomedicine, it is essential to …

Synthesis, Biophysical and Biological Evaluation of Splice-Switching Oligonucleotides with Multiple LNA-Phosphothiotriester Backbones

D Dhara, AC Hill, A Ramesh, MJA Wood… - Journal of the …, 2024 - ACS Publications
Polyanionic antisense oligonucleotides hold great promise as RNA targeting drugs but
issues with bioavailability hinder their development. Uncharged phosphorus-based …

2′-O-Alkyl-N3-Methyluridine Functionalized Passenger Strand Improves RNAi Activity by Modulating the Thermal Stability

A Sahoo, S Gupta, G Das, A Ghosh… - ACS Medicinal …, 2024 - ACS Publications
Herein, we have demonstrated that the siRNA activity could be enhanced by incorporating
the guide strand in the RISC complex through thermodynamic asymmetry caused by m3U …