Results 51 to 60 of about 94,605 (281)

Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs

Advanced Healthcare Materials, EarlyView.
Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner, Stefan Keim, Soheil Haddadzadegan, Odile Fernandez Romero, Katrin Zöller, Gabriel Stern, Ilaria Cesi, Krum Kafedjiiski, Andreas Bernkop‐Schnürch   +8 more
wiley   +1 more source

Nonviral delivery systems for antisense oligonucleotide therapeutics

Biomaterials Research, 2022
Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang, Xin-Yan Hao, Yong-Jiang Li, Jun‑Yong Wu, Da-Xiong Xiang, Shilin Luo   +5 more
doaj   +1 more source

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy

Nature Communications, 2023
In a double homozygous mouse model of myotonic dystrophy type 1, Hu et al. use antisense oligonucleotide correction of myotonia to induce a therapeutic shift from an overabundance of oxidative muscle fibers to mechanically stronger glycolytic fibers.
Ningyan Hu, Eunjoo Kim, Layal Antoury, Thurman M. Wheeler   +3 more
doaj   +1 more source

CCDC41 Drives Oocyte Meiotic Progression by Promoting Rab11a/Rab7‐Positive Vesicle Fusion with Target Membranes

Advanced Science, EarlyView.
CCDC41 is essential for meiotic maturation in mouse oocytes through regulating Rab7‐positive endosomes fusion with lysosomes and Rab11a‐positive vesicle fusion with the plasma membrane. Abstract Coiled‐coil domain‐containing protein 41 (CCDC41), a core component of centriolar distal appendages involved in centriole assembly and ciliary vesicle docking,
Ying Tian, Jiatong Li, Jingyi Kang, Xiangning Xu, Bicheng Wang, Shuo Lou, Jingyu Li, Yuying Yang, Yanbing Zhang, Yangzi Zheng, Jing Weng, Yuanjing Liang, Wei Ma   +12 more
wiley   +1 more source

A Unique Chimeric RNA: ERCC1‐iASPP Drives Benzo[a]pyrene‐Induced Lung Carcinogenesis via Dual Coding and Non‐Coding Mechanisms

Advanced Science, EarlyView.
The chimeric RNA ERCC1‐iASPP possesses dual coding and non‐coding functions, synergistically accelerating the process of cellular malignant transformation. Abstract Genetic variation at 19q13.3 critically modulates chemical carcinogen‐induced lung carcinogenesis, particularly in mediating the activity of benzo[a]pyrene (B[a]P), a major polycyclic ...
Mingming Shan, Mingyang Xiao, Liang Zhang, Shen Wang, Jiahang Wu, Kang Cao, Yujin Li, Guopei Zhang, Junwei Xie, Xiaobo Lu   +9 more
wiley   +1 more source

Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile

International Journal of Nanomedicine, 2016
John P Hegarty,1 Jacek Krzeminski,2 Arun K Sharma,2 Diana Guzman-Villanueva,3 Volkmar Weissig,3 David B Stewart Sr1 1Deparment of Surgery, Pennsylvania State University College of Medicine Hershey, PA, USA; 2Department of Pharmacology, Penn State ...
Hegarty JP, Krzeminski J, Sharma AK, Guzman-Villanueva D, Weissig V, Stewart DB Sr   +5 more
doaj  

Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy

Nature Communications, 2022
The W1282X nonsense mutation in the CFTR gene causes cystic fibrosis by reducing its mRNA and functional protein levels. Here the authors developed antisense-oligonucleotide cocktails that restore CFTR protein function by gene-specific stabilization of ...
Young Jin Kim, Tomoki Nomakuchi, Foteini Papaleonidopoulou, Lucia Yang, Qian Zhang, Adrian R. Krainer   +5 more
doaj   +1 more source

USP9X as a Candidate Mediator of Prenatal Aspirin‐Induced Ovarian Reserve Reduction in Offspring Mice

Advanced Science, EarlyView.
This study suggests that prenatal aspirin exposure is associated with reduced ovarian reserve in offspring, associated with HDAC1‐linked epigenetic downregulation of Usp9x as a candidate mechanism. These preclinical findings provide new insights into fetal‐origin ovarian disorders and contribute to the evidence base concerning aspirin's gestational ...
Yating Li, Caiyun Ge, Wai Yen Yim, Hui Feng, Tiancheng Wu, Lu Chen, Qiaohua Xiong, Shumin Pan, Mei Wang, Huijun Chen, Yuanzhen Zhang, Hui Wang   +11 more
wiley   +1 more source

When HERG-caused LQT2 encounters antisense oligonucleotide: is exon 6 skipping therapy plausible?

Frontiers in Pharmacology
Graphical AbstractThe unique in‐frame exon 6 of the HERG gene as a potential target for antisense oligonucleotide‐mediated exon skipping therapy.
Zequn Zheng, Zequn Zheng, Yongfei Song
doaj   +1 more source