Results 221 to 230 of about 263,255 (408)

Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen‐Treated ALS

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)‐ALS patients treated with the antisense oligonucleotide tofersen show a ...
Sarah Bernsen, Rachel Fabian, Yasemin Koc, Peggy Schumann, Peter Körtvélyessy, Sergio Castro‐Gomez, Thomas Meyer, Patrick Weydt   +7 more
wiley   +1 more source

Inhibition of EGF Uptake by Nephrotoxic Antisense Drugs In Vitro and Implications for Preclinical Safety Profiling

Molecular Therapy: Nucleic Acids, 2017
Antisense oligonucleotide (AON) therapeutics offer new avenues to pursue clinically relevant targets inaccessible with other technologies. Advances in improving AON affinity and stability by incorporation of high affinity nucleotides, such as locked ...
Annie Moisan, Marcel Gubler, Jitao David Zhang, Yann Tessier, Kamille Dumong Erichsen, Sabine Sewing, Régine Gérard, Blandine Avignon, Sylwia Huber, Fethallah Benmansour, Xing Chen, Roberto Villaseñor, Annamaria Braendli-Baiocco, Matthias Festag, Andreas Maunz, Thomas Singer, Franz Schuler, Adrian B. Roth   +17 more
doaj  

Single intraluminal delivery of antisense cdc2 kinase and proliferating-cell nuclear antigen oligonucleotides results in chronic inhibition of neointimal hyperplasia.

, 1993
Ryuichi Morishita, Gary H. Gibbons, Kristin Ellison, Masatoshi Nakajima, L Zhang, Yudai Kaneda, Toshio Ogihara, Victor J. Dzau   +7 more
openalex   +1 more source

Progress in Aptamer Research and Future Applications

ChemistryOpen, EarlyView.
This paper mainly describes the screening method of aptamer, the screening difficulties and improvement methods of small molecule aptamer, and the application and treatment of aptamer. It explores the latest progress and application of aptamers, and summarises them, thus indicating some problems that still exist in this field.
Song Liu, Xiaolu Li, Huyang Gao, Jing Chen, Hongfeng Jiang   +4 more
wiley   +1 more source

Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides [PDF]

, 1999
Kenneth J. Friedman, Jolanta Kole, Jonathan Cohn, Michael R. Knowles, Lawrence M. Silverman, Ryszard Kole   +5 more
openalex   +1 more source

Update on Gene Therapy in the Treatment of Hereditary Hearing Loss

Sensory Neuroscience, EarlyView.
Gene therapy is a promising therapeutic approach for genetic disorders, including three major gene therapy strategies: gene replacement, gene suppression, and gene editing. After transmitting genes to the inner ear through appropriate pathways, repair the cochlea in order to achieve the goal of treating hereditary hearing loss.
Weiwei He, Shanying Han, Xinghong Liu, Xiaotong Huang, Wen Yang, Xinghua Tang, Chuan Dong, Jiangang Fan   +7 more
wiley   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

NIR‐Induced Photoswitching Hybrid DNA Nanoconstruct‐Based Drug Delivery System for Spatiotemporal Control of Stem Cell Fate

Small, EarlyView.
NIR‐mediated photo‐switching drug delivery system for the remote‐control differentiation of NSCs. Left: NIR‐mediated curcumin release is induced by NIR‐to‐UV photon upconversion in a photo‐controllable manner. Curcumin is bound in the minor groove of the synthesized DNA construct containing AAP photo‐switchers. Right: The rational design of several AAP
Thanapat Pongkulapa, Ji Hye Yum, Callan D. McLoughlin, Brandon Conklin, Tomotaka Kumagai, Li Ling Goldston, Hiroshi Sugiyama, Soyoung Park, Ki‐Bum Lee   +8 more
wiley   +1 more source

Nucleic Acid‐Modified Nanoparticles for Cancer Therapeutic Applications

Small, EarlyView.
Nucleic acid‐modified nanoparticles act as functional hybrids for chemodynamic, photodynamic, photothermal, and gene therapy of cancer cells. These are exemplified with aptamer‐modified catalytic nanoparticles, photosensitizer‐oligonucleotide functionalized nanoparticles, drug‐loaded stimuli‐responsive nucleic acid‐caged nanoparticle carriers, and ...
Yunlong Qin, Xinghua Chen, Itamar Willner   +2 more
wiley   +1 more source

Systemic Brain Delivery of Oligonucleotide Therapeutics Enhanced by Protein Corona‐Assisted DNA Cubes

Small Methods, EarlyView.
In vivo, screening of DNA nanostructures yields a cube‐shaped structure (D‐Cb) as a protein corona‐assisted platform for brain delivery of oligonucleotide therapeutics. D‐Cb effectively delivers polo‐like kinase 1 (PLK‐1) antisense oligonucleotides (ASO) to treat glioblastoma in an orthotopic mouse model, demonstrating its potential as a carrier for ...
Kyoung‐Ran Kim, Ji Hee Kang, Hien Bao Dieu Thai, Ji Hyun Back, Chengde Mao, Ji Eun Lee, Young Tag Ko, Dae‐Ro Ahn   +7 more
wiley   +1 more source