Results 31 to 40 of about 45,567 (253)

A mechanistic pharmacokinetic model for intrathecal administration of antisense oligonucleotides

Frontiers in Physiology, 2023
Intrathecal administration is an important mode for delivering biological agents targeting central nervous system (CNS) diseases. However, current clinical practices lack a sound theorical basis for a quantitative understanding of the variables and ...
Andreas A. Linninger, Dipak Barua, Yaming Hang, Sergio Iadevaia, Majid Vakilynejad   +4 more
doaj   +1 more source

Purification and preparation of Marchantia polymorpha Auxin Response Factor 2 for phase separation studies

FEBS Open Bio, EarlyView.
We describe detailed protocols for the purification and preparation of Marchantia polymorpha Auxin Response Factor 2 (MpARF2). This protein is fused to an MBP solubility tag and an mNG fluorescent tag and is purified from Escherichia coli. The presented procedures make it possible to study MpARF2 assemblies, which could arise from phase separation ...
Bas Janssen, Robin Romein, Willy A. M. van den Berg, Simon Lindhoud, Jan Willem Borst, Carlo P. M. van Mierlo   +5 more
wiley   +1 more source

Fusogenic RNA Nanomodules for Fusion‐Mediated and Multiplexed siRNA Delivery

Advanced Functional Materials, EarlyView.
A fusogenic lipid‐layered RNA nanomodules (L‐CRAMs) enable high‐capacity and long‐lasting siRNA delivery through membrane fusion. These nanomodules carry exceptionally large siRNA payloads, avoid conventional endosomal uptake, and release multiple functional siRNAs through Dicer‐mediated processing.
Sunghyun Moon, Iksoo Jang, Taehyeon Kim, Nayoung Yoon, Kyuha Yum, Jong Bum Lee   +5 more
wiley   +1 more source

Efficient Modulation of Exon Skipping via Antisense Circular RNAs

Research, 2023
Splice-switching antisense oligonucleotides (ASOs) and engineered U7 small nuclear ribonucleoprotein (U7 Sm OPT) are the most commonly used methods for exon skipping. However, challenges remain, such as limited organ delivery and repeated dosing for ASOs
Shuaiwei Ren, Mei Huang, Raoxian Bai, Lijiao Chen, Jiao Yang, Junyu Zhang, Wenting Guo, Weizhi Ji, Yongchang Chen   +8 more
doaj   +1 more source

Antisense delivery using protamine-oligonucleotide particles [PDF]

Nucleic Acids Research, 2000
Protamine, a polycationic peptide (mol. wt 4000-4500), was evaluated as a potential penetration enhancer for phosphodiester antisense oligonucleotides (ODNs). Unique complexes in the form of nanoparticles were spontaneously formed, which we call 'proticles'.
M, Junghans, J, Kreuter, A, Zimmer
openaire   +2 more sources

Nanomaterial Strategies for Pulmonary Delivery of Immunotherapeutics in Lung Cancer Treatment

Advanced Healthcare Materials, EarlyView.
Inhalable immunotherapeutic nanomedicines enable organ‐selective immune modulation by overcoming pulmonary delivery barriers and concentrating therapy within lung tumors. This Review defines how nanomaterial properties govern airway deposition, retention, cellular partitioning, and immune activation across vaccines, checkpoint blockade, STING agonists,
Han Zhang, Wei Tang
wiley   +1 more source

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Molecular Therapy: Nucleic Acids, 2017
Antisense-mediated exon skipping is a promising approach for the treatment of Duchenne muscular dystrophy (DMD), a rare life-threatening genetic disease due to dystrophin deficiency.
Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell, Fred Schnell, Gunnar Hanson, George Dickson   +5 more
doaj   +1 more source

Multifunctional Thiolated Hyaluronic Acid‐graft‐PEDOT Dressing With Antifouling, On‐Demand Drug Delivery and Electrical Stimulation for Wound Healing

Advanced Healthcare Materials, EarlyView.
A multifunctional P(EDOT‐co‐EDOT‐g‐THA) dressing acts as a dynamic biointerface integrating antifouling capability, on‐demand drug delivery, and electrical stimulation. It achieves electrochemically triggered thiolated hyaluronic acid (THA) release while modulating cellular responses for wound healing application.
Jingwen Yang, Jiwon Hong, Anthony Phillips, Bicheng Zhu, Jenny Malmstrom, Devon T. Bryant, Alireza Akbarinejad, Lisa I. Pilkington, Jadranka Travas‐Sejdic   +8 more
wiley   +1 more source

Gene and Antisense Delivery in Alcoholism Research

Alcoholism: Clinical and Experimental Research, 2002
This article represents the proceedings of a symposium at the 2001 annual meeting of the Research Society on Alcoholism in Montreal, Canada. Drs. Yedy Israel and Fulton Crews were organizers and co‐chairpersons. The presentations were (1) Introduction to the symposium, by Yedy Israel; (2) Gene delivery to the brain, by Fulton T. Crews; (3) Gene therapy
Israel Jacard, Yedy, Crews, F. T., Thurman, R. G., Tu, G. C., Garver, E., Ponnappa, B., Karahanian, E., Rubin, R., Hoplight, B., Sethna, M., Hanes, R., Wilkie, M. B., Wheeler, M. D.   +12 more
openaire   +3 more sources

Negotiating in a Foreign Land: Understanding the Curious Interactions Between Intracellular Mitochondria and Internalized Nanoparticles

Advanced Materials Interfaces, EarlyView.
Therapeutic nano‐drug delivery systems interact with cellular mitochondria in a multitude of ways. While the complexity of such interactions disrupts the mitochondrial electron transport chain and increases reactive oxygen species production, thereby contributing to nanoparticle toxicity, they also present unique theranostic opportunities in diseases ...
Sourav Bhattacharjee
wiley   +1 more source