Results 61 to 70 of about 171,175 (241)

Repair of Aberrant Splicing in Growth Hormone Receptor by Antisense Oligonucleotides Targeting the Splice Sites of a Pseudoexon [PDF]

, 2010
Context: The GH receptor (GHR) pseudoexon 6 Psi defect is a frequent cause of GH insensitivity (GHI) resulting from a non-functioning GH receptor (GHR). It results in a broad range of phenotypes and may also be present in patients diagnosed as idiopathic
Srirangalingam, U, David, A, Metherell, LA, Khoo, B, Clark, AJL   +4 more
core  

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  

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

Unraveling Bone‐Skin Crosstalk Enables miRNA Nanoformulation for Cutaneous Neurovascular Reconstruction in Diabetic Mice

Advanced Science, EarlyView.
This study unravels that bone fracture accelerates diabetic wound healing by releasing exosomal microRNA (miR‐130b‐3p). To mimic this bone‐skin crosstalk, we engineered a self‐assembling agomir‐130b‐3p nanocomplex. Delivered via a photocrosslinkable hydrogel, this bio‐inspired therapy provides sustained localized agomir release.
Tao Shen, Hongdou Shen, Weiyue Wu, Zhuoying Jiang, Bin Liu, Yansi Xian, Rui Peng, An Yan, Yu Ben, Yong Shi, Xiang Chen, Baosheng Guo, Qing Jiang, Weijun Wang, Pingqiang Cai   +14 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

Single‐cell Transcriptome Profiling Reveals Gene Regulatory Networks and Key Genes in the Root Epidermis and Cortical Cells Associated with Early Nodulation in Glycine Max

Advanced Science, EarlyView.
Single‐cell transcriptomics of soybean roots soon after rhizobial inoculation reveals epidermal and cortical cell‐specific programs and gene‐regulatory networks acting in symbiosis establishment. We identify an ethylene‐driven regulatory circuit involving WRKY6.3/6.4 transcription factors targeting select Nod19 genes that promotes infection‐thread ...
Yongbin Zhuang, Yu Geng, Xinyu Guo, Chen Wang, Yongjing Sun, Luqi Liu, Li Liu, QianQian Gao, XinFang Zhu, Hongxiang Cao, Gengshuo Zhang, Yunxia Liu, Guobin Zhang, Baoyin Chen, Jinfei Zhang, Xin Hou, Xiaoming Li, Dajian Zhang   +17 more
wiley   +1 more source

ANTISENSE MEDIATED DYSTROPHIN READING FRAME RESTORATION [PDF]

, 2010
Exon skipping using antisense oligonucleotides (AONs) has successfully been used to reframe the mRNA in various DMD (Duchenne muscular dystrophy) patients carrying deletions and in the mdx mouse model.
Spitali, Pietro
core  

Cell‐Selective Delivery of RIBOTACs via an Anti‐EGFR Nanobody for Pancreatic Cancer Treatment

Advanced Science, EarlyView.
This study introduces an innovative strategy for the tumor‐selective catalytic degradation of oncogenic non‐coding RNA by interfacing a ribonuclease‐recruiting small molecule (RIBOTAC) with an EGFR‐targeting nanobody via a CTSB (Cathepsin B)‐responsive linker.
Tianli Luo, Yijuan Wang, Dengwang Chen, Yinghan Zhang, Yi Shuai, Hualiang Yao, Tao Jiang, Hang Jiang, Ming Zhong, Xiangeng Wang, Ziwei Wang, Chenchen Xu, Ajay Goel, Hoi Tang MA, Feng Gao, Xin Wang   +15 more
wiley   +1 more source

Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis

Annals of Clinical and Translational Neurology
Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been ...
Daisuke Ito, Kensuke Okada
doaj   +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