Results 81 to 90 of about 275,477 (365)

Site Selective Antibody-Oligonucleotide Conjugation via Microbial Transglutaminase. [PDF]

, 2019
Nucleic Acid Therapeutics (NATs), including siRNAs and AntiSense Oligonucleotides (ASOs), have great potential to drug the undruggable genome. Targeting siRNAs and ASOs to specific cell types of interest has driven dramatic improvement in efficacy and ...
Cui, Xianshu, Dowdy, Steven F, Huggins, Ian J, Jadhav, Satish, Medina, Carlos A, Springer, Aaron D, van den Berg, Arjen   +6 more
core   +1 more source

Rewiring Neuroimmunity: Nanoplatform Innovations for CNS Disease Therapy

Advanced Therapeutics, EarlyView.
This review explores emerging nanoplatform strategies designed to modulate neuroimmune responses for treating central nervous system (CNS) disorders. It examines structural and microenvironmental barriers, advances in multifunctional and targeted nanotechnologies, and highlights clinical progress and translational challenges, offering insights into the
Muhammad Usman Akbar, Weiwei Guo, Xin Shen, Yimeng Fang, Yilin Liu, Peng‐Yuan Wang, Roey Elnathan, Yaping Chen   +7 more
wiley   +1 more source

Recent Advancements in Development and Therapeutic Applications of Genome-Targeting Triplex-Forming Oligonucleotides and Peptide Nucleic Acids

Pharmaceutics, 2023
Recent developments in artificial nucleic acid and drug delivery systems present possibilities for the symbiotic engineering of therapeutic oligonucleotides, such as antisense oligonucleotides (ASOs) and small interfering ribonucleic acids (siRNAs ...
Yu Mikame, Asako Yamayoshi
doaj   +1 more source

Emerging Therapeutic Strategies for Hearing Loss

Advanced Therapeutics, EarlyView.
Challenges still exist in treating hearing loss in cases of severe damage to hair cells or spiral ganglion neurons. Here, a schematic diagram of cochlear sensory hair cells and auditory nerves is presented. It is found that in normal Corti organs, hair cells have upright stereocilia at the top and auditory neurons at the base. Ototoxic drugs, noise, or
Shanying Han, Tian Shen, Weiwei He, Hao Wei, Xiaolong Zhao, Wen Yang, Chuan Bu, Xinghua Tang, Yu Zhao, Jiangang Fan   +9 more
wiley   +1 more source

Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver.

Nucleic Acid Therapeutics, 2018
Oligonucleotide therapeutics have emerged as a third distinct platform for drug discovery within the pharmaceutical industry. Five oligonucleotide-based drugs have been approved by the US FDA and over 100 oligonucleotides drugs are currently at different
Colton M. Miller, M. Tanowitz, A. J. Donner, T. P. Prakash, E. Swayze, E. Harris, P. Seth   +6 more
semanticscholar   +1 more source

Long‐Lasting Auditory and Vestibular Recovery Following Gene Replacement Therapy in a Novel Usher Syndrome Type 1c Mouse Model

Advanced Science, EarlyView.
This study shows that gene replacement therapy using the AAV2/Anc80L65 virus can successfully restore hearing and balance in Ush1c knockout mice. The treatment leads to lasting improvements in both auditory and vestibular functions, highlighting its potential as a therapeutic approach for genetic hearing loss and vestibular disorders in humans ...
Weinan Du, Jun Huang, Aizhen Zhang, Fangfang Zhao, Tianwen Chen, Quinn M. McDermott, Tony Zheng, Haibo Wang, Rongli Zhang, Xiaolin Zhang, Jerome Allison, Hong Zhu, Wu Zhou, Qing Yin Zheng   +13 more
wiley   +1 more source

Making Sense of Antisense

Canadian Journal of Gastroenterology, 1999
Since the identification of the DNA double-stranded helix, the gene as a target of therapy and, moreover, the use of DNA as a drug have been possibilities.
Bruce R Yacyshyn, William R Shanahan
doaj   +1 more source

Antisense oligonucleotide induction of progerin in human myogenic cells. [PDF]

PLoS ONE, 2014
We sought to use splice-switching antisense oligonucleotides to produce a model of accelerated ageing by enhancing expression of progerin, translated from a mis-spliced lamin A gene (LMNA) transcript in human myogenic cells. The progerin transcript (LMNA
Yue-Bei Luo, Chalermchai Mitrpant, Abbie M Adams, Russell D Johnsen, Sue Fletcher, Frank L Mastaglia, Steve D Wilton   +6 more
doaj   +1 more source

Rational Design of Inner Ear Drug Delivery Systems

Advanced Science, EarlyView.
Hearing loss is a common disease affecting many people, and inner ear lesions are one of the most important causes. This review focuses on the treatment of inner ear hearing loss by drug delivery systems. It includes the current methods and technologies developed, and it predicts possible directions.
Xiayidan Maimaitikelimu, Zhiyan Xuan, Haoyu Ren, Keng Chen, Hui Zhang, Huan Wang   +5 more
wiley   +1 more source

Formation of an RNA Quadruplex-Duplex Hybrid in Living Cells between mRNA of the Epidermal Growth Factor Receptor (EGFR) and a G-Rich Antisense Oligoribonucleotide

Cells, 2020
Antisense DNA oligonucleotides, short interfering RNAs (siRNAs), and CRISPR/Cas9 genetic tools are the most useful therapeutic nucleic acids regulating gene expression based on the antisense specificity towards messenger RNA.
Dorota Gudanis, Damian Kaniowski, Katarzyna Kulik, Daniel Baranowski, Zofia Gdaniec, Barbara Nawrot   +5 more
doaj   +1 more source