Exploring RNA therapeutics for urea cycle disorders
E Richard, A Martínez‐Pizarro… - Journal of Inherited …, 2024 - Wiley Online Library
RNA has triggered a significant shift in modern medicine, providing a promising way to
revolutionize disease treatment methods. Different therapeutic RNA modalities have shown …
revolutionize disease treatment methods. Different therapeutic RNA modalities have shown …
MicroRNAs in metabolic dysfunction‐associated diseases: Pathogenesis and therapeutic opportunities
N Ma, J Tan, Y Chen, L Yang, M Li, Y He - The FASEB Journal, 2024 - Wiley Online Library
Metabolic dysfunction‐associated diseases often refer to various diseases caused by
metabolic problems such as glucose and lipid metabolism disorders. With the improvement …
metabolic problems such as glucose and lipid metabolism disorders. With the improvement …
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
R Miller, J Paquette, A Barker, E Sapp… - … Therapy Nucleic Acids, 2024 - cell.com
Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of
gene expression in the central nervous system (CNS), with several drugs approved and …
gene expression in the central nervous system (CNS), with several drugs approved and …
Why and How to Control P-Chirality in Phosphorothioated Therapeutic Oligonucleotides: Analytical Challenges Associated with Determination of Stereochemical …
T Poredoš, M Trampuž, T Gornik… - … Process Research & …, 2025 - ACS Publications
A surge in the approval of therapeutic oligonucleotides since 2016 has made this class of
chemical modalities an essential toolbox for addressing unmet medical needs in the realm …
chemical modalities an essential toolbox for addressing unmet medical needs in the realm …
Molecular Dynamics Simulations Elucidate the Molecular Organization of Poly (beta-amino ester) Based Polyplexes for siRNA Delivery
KM Steinegger, L Allmendinger, S Sturm… - Nano Letters, 2024 - ACS Publications
Cationic polymers are known to efficiently deliver nucleic acids to target cells by
encapsulating the cargo into nanoparticles. However, the molecular organization of these …
encapsulating the cargo into nanoparticles. However, the molecular organization of these …
[HTML][HTML] Therapeutic oligonucleotide ASC1R shows excellent tolerability and remarkable efficacy in reducing SARS-CoV-2 mRNA levels in C57BL/6 mice
V Nemethova, P Babiakova, M Selc, K Jakic… - Biomedicine & …, 2024 - Elsevier
The coronavirus pandemic has resulted in over 775 million cases and 7 million deaths
worldwide, driving efforts to develop therapeutic strategies to control the viral infection …
worldwide, driving efforts to develop therapeutic strategies to control the viral infection …
MicroRNAs and their Modulatory Effect on the Hallmarks of Osteosarcopenia
Abstract Purpose of the Review Osteosarcopenia is a geriatric syndrome associated with
disability and mortality. This review summarizes the key microRNAs that regulate the …
disability and mortality. This review summarizes the key microRNAs that regulate the …
Hepatocyte targeting via the asialoglycoprotein receptor
F Ramírez-Cortés, P Ménová - RSC Medicinal Chemistry, 2024 - pubs.rsc.org
This review highlights the potential of asialoglycoprotein receptor (ASGPR)-mediated
targeting in advancing liver-specific treatments and underscores the ongoing progress in the …
targeting in advancing liver-specific treatments and underscores the ongoing progress in the …
Applications of Hydrophilic Interaction and Mixed-mode Liquid Chromatography in Pharmaceutical Analysis
B Wei, L Dai, K Zhang - Journal of Chromatography A, 2024 - Elsevier
Abstract Hydrophilic Interaction Liquid Chromatography (HILIC) and Mixed-Mode
Chromatography (MMC) excel in separating polar, hydrophilic, and charged analytes due to …
Chromatography (MMC) excel in separating polar, hydrophilic, and charged analytes due to …
Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges
R Ruchi, GM Raman, V Kumar… - Expert Opinion on Drug …, 2025 - Taylor & Francis
Introduction Antisense oligonucleotide (ASO) was established as a viable therapeutic option
for genetic disorders. ASOs can target RNAs implicated in various diseases, including …
for genetic disorders. ASOs can target RNAs implicated in various diseases, including …