Results 11 to 20 of about 1,703,096 (348)

Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies

Biomedicines, 2021
The literature surrounding the use of antisense oligonucleotides continues to grow, with new disease and mechanistic applications constantly evolving. Furthermore, the discovery and advancement of novel chemistries continues to improve antisense delivery,
Loren L. Flynn, Chalermchai Mitrpant, Abbie Adams, Ianthe L. Pitout, Anja Stirnweiss, Sue Fletcher, Steve D. Wilton   +6 more
doaj   +1 more source

Versatility of Liposomes for Antisense Oligonucleotide Delivery: A Special Focus on Various Therapeutic Areas

Pharmaceutics, 2023
Nucleic acid therapeutics, specifically antisense oligonucleotides (ASOs), can effectively modulate gene expression and protein function, leading to long-lasting curative effects.
Raghav Gupta, Sagar Salave, Dhwani Rana, Bharathi Karunakaran, Arun Butreddy, Derajram Benival, Nagavendra Kommineni   +6 more
semanticscholar   +1 more source

Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice

Nucleic Acids Research, 2022
Current therapies for Duchenne muscular dystrophy (DMD) use phosphorodiamidate morpholino oligomers (PMO) to induce exon skipping in the dystrophin pre-mRNA, enabling the translation of a shortened but functional dystrophin protein.
Cody A. Desjardins, M. Yao, John Hall, Emma O'Donnell, Reshmii Venkatesan, S. Spring, Aiyun Wen, Nelson Hsia, Peiyi Shen, R. Russo, Bo Lan, T. Picariello, Kim Tang, T. Weeden, S. Zanotti, Romesh R. Subramanian, O. Ibraghimov-Beskrovnaya   +16 more
semanticscholar   +1 more source

Advances in the Design of (Nano)Formulations for Delivery of Antisense Oligonucleotides and Small Interfering RNA: Focus on the Central Nervous System

Molecular Pharmaceutics, 2021
RNA-based therapeutics have emerged as one of the most powerful therapeutic options used for the modulation of gene/protein expression and gene editing with the potential to treat neurodegenerative diseases.
M. C. Mendonça, Ayse Kont, M. R. Aburto, J. Cryan, C. O’Driscoll   +4 more
semanticscholar   +1 more source

iRGD-liposomes enhance tumor delivery and therapeutic efficacy of antisense oligonucleotide drugs against primary prostate cancer and bone metastasis

bioRxiv, 2021
Nucleotide-based drugs, such as antisense oligonucleotides (ASOs), have unique advantages in treating human diseases as they provide virtually unlimited ability to target any gene.
Jibin Guan, Hong Guo, Tang Tang, Yihan Wang, Yushuang Wei, P. Seth, Yingming Li, S. Dehm, E. Ruoslahti, Hong‐Bo Pang   +9 more
semanticscholar   +1 more source

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review

European Journal of Cell Biology, 2023
Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
doaj   +1 more source

Self-transfecting GMO-PMO chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse

Molecular Therapy: Nucleic Acids, 2023
Phosphorodiamidate morpholino oligonucleotide (PMO)-based antisense reagents cannot enter cells without the help of a delivery technique, which limits their clinical applications. To overcome this problem, self-transfecting guanidinium-linked morpholino (
Ujjal Das, Jayanta Kundu, Pallab Shaw, Chandra Bose, Atanu Ghosh, Shalini Gupta, Sudipta Sarkar, Jhuma Bhadra, Surajit Sinha   +8 more
doaj   +1 more source

Selected strategies to fight pathogenic bacteria

Journal of Enzyme Inhibition and Medicinal Chemistry, 2023
Natural products and analogues are a source of antibacterial drug discovery. Considering drug resistance levels emerging for antibiotics, identification of bacterial metalloenzymes and the synthesis of selective inhibitors are interesting for ...
Aiva Plotniece, Arkadij Sobolev, Claudiu T. Supuran, Fabrizio Carta, Fredrik Björkling, Henrik Franzyk, Jari Yli-Kauhaluoma, Koen Augustyns, Paul Cos, Linda De Vooght, Matthias Govaerts, Juliana Aizawa, Päivi Tammela, Raivis Žalubovskis   +13 more
doaj   +1 more source

A Novel Targeted Therapy System for Cervical Cancer: Co-Delivery System of Antisense LncRNA of MDC1 and Oxaliplatin Magnetic Thermosensitive Cationic Liposome Drug Carrier

International Journal of Nanomedicine, 2021
Background This study was aimed to prepare a novel magnetic thermosensitive cationic liposome drug carrier for the codelivery of Oxaliplatin (OXA) and antisense lncRNA of MDC1 (MDC1-AS) to Cervical cancer cells and evaluate the efficiency of this drug ...
Hui Ye, Xiaoying Chu, Z. Cao, Xuanxuan Hu, Zihan Wang, Meiqi Li, Leyu Wan, Yongping Li, Yongge Cao, Zhanqiu Diao, Fengting Peng, Jinsong Liu, Lihua Xu   +12 more
semanticscholar   +1 more source

A Self-assembled Platform Based on Branched DNA for sgRNA/Cas9/Antisense Delivery.

Journal of the American Chemical Society, 2019
Precisely assembled DNA nanostructures are promising candidates for the delivery of biomolecule-based therapeutics. Herein, we introduce a facile strategy for the construction of a branched DNA-based nanoplatform for co-delivery of gene editing (sgRNA ...
Jianbing Liu, Tiantian Wu, Xuehe Lu, Xiaohui Wu, Shaoli Liu, Shuai Zhao, Xuehui Xu, Baoquan Ding   +7 more
semanticscholar   +1 more source