Qfitlia (Fitusiran): A Novel siRNA-based Treatment for Haemophilia  

Qfitlia (Fitusiran), a novel siRNA-based treatment for haemophilia has received FDA approval. It is a small interfering RNA (siRNA) based therapeutic

Qfitlia (Fitusiran): A Novel siRNA-based Treatment for Haemophilia  

Qfitlia (Fitusiran), a novel siRNA-based treatment for haemophilia has received FDA approval. It is a small interfering RNA (siRNA) based therapeutic

Protecting crops: RNA-based substances open up new avenue to combat a widespread plant virus

New RNA-based active agents reliably protect plants against the Cucumber mosaic virus (CMV), the most common virus in agriculture and horticulture. They were developed by researchers at the Martin Luther University Halle-Wittenberg (MLU).

Nanotechnology - Computational Approaches in Improving Spermine-based RNA Nanocarriers - Beilstein-Institut

Nanotechnology - Computational Approaches in Improving Spermine-based RNA Nanocarriers - Beilstein-Institut

Nanotechnology - Computational Approaches in Improving Spermine-based RNA Nanocarriers - Beilstein-Institut

Dall'Univaq una cura per l'osteoporosi autosomica

Brevettato all'Aquila farmaco contro malattia rara delle ossa. Dall'Università è stato dato in licenza a un'azienda britannica e ora è in attesa del via l

Current State of Oligonucleotide Therapeutics

Oligonucleotides are a relatively new class of drugs, composed of natural and synthetic nucleotides, which primarily include small interfering RNA (siRNA), micro RNA (miRNA), and antisense oligonucleotide (ASO). These molecules achieve therapeutic effects through RNA interference, degradation, or splice-modulating pathways.Weng, Y., H. Xiao, J. Zhang, X. J. Liang, and Y. Huang. “RNAi Therapeutic and Its Innovative Biotechnological Evolution.” Biotechnology Advances 37, no. 5 (2019): 801–825., Setten, R. L., J. J. Rossi, and S. P. Han. “The Current State and Future Directions of RNAi-Based Therapeutics.” Nature Reviews Drug Discovery 18, no. 6 (2019): 421–446., Wang, F., T. Zuroske, and J. K. Watts. “RNA Therapeutics on the Rise.” Nature Reviews Drug Discovery 19, no. 7 (2020): 441–442. doi:10.1038/d41573-020-00078-0, Hu, B., L. Zhong, Y. Weng, et al. “Therapeutic siRNA: State of the Art.” Signal Transduction and Targeted Therapy 5, no. 1 (2020): 101. https://www.nature.com/articles/s41392-020-0207-x Other oligonucleotide therapies include messenger RNA (mRNA, single strand, >500 mers),Weng, Y., C. Li, T. Yang, et al. “The Challenge and Prospect of mRNA Therapeutics Landscape.” Biotechnology Advances 40 (2020): 107534. small activation RNA (double strand, ~20 mers),Li, L. C., S. T. Okino, H. Zhao, et al. “Small dsRNAs Induce Transcriptional Activation in Human Cells.” Proceedings of the National Academies of Sciences of the Unites States of America 103, no. 46 (2006): 17337–17342. antagomir (single strand, ~20 mers),Innao, V., A. Allegra, N. Pulvirenti, A. G. Allegra, and C. Musolino. “Therapeutic Potential of antagomiRs in Haematological and Oncological Neoplasms. European Journal of Cancer Care 29, no. 2 (2020): e13208. and aptamer (single strand, >30 mers).Zhou, J., and J. Rossi. “Aptamers as Targeted Therapeutics: Current Potential and Challenges.” Nature Reviews Drug Discovery 16 (2017): 181–202. This article focuses on synthetic ASOs (single strand, 16–22 mers) and siRNAs (double strands formed by hybridization of a pair of complementary sense and antisense strands, 19–25 mers).