Results 111 to 120 of about 240,839 (324)

Identification of NIBAN2‐Regulated RUNX2 Alternative Splicing Presents Novel Strategies for Antagonizing Osteoporosis

Advanced Science, EarlyView.
NIBAN2 interacts with the HNRNPU‐cored spliceosome complex and alters its components to regulate the alternative splicing of RUNX2, which ultimately cause an increase in functional RUNX2 (nuclear localization sequence complete) but a decrease in dysfunctional Runx2 (exon 6‐exclusive) isoforms to reinforce osteoblast differentiation.
Sheng Zhang, Zhiqiang Yang, Yuanlong Xie, Yufeng Zhang, Zhe Chen, Xuan Lv, Zhouming Deng, Zan Huang, Lin Cai, Renxiong Wei   +9 more
wiley   +1 more source

Molecular mechanisms of Eda‐mediated adaptation to freshwater in threespine stickleback

Molecular Ecology, EarlyView., 2023
Abstract A main goal of evolutionary biology is to understand the genetic basis of adaptive evolution. Although the genes that underlie some adaptive phenotypes are now known, the molecular pathways and regulatory mechanisms mediating the phenotypic effects of those genes often remain a black box.
Carlos E. Rodríguez‐Ramírez, Melanie Hiltbrunner, Verena Saladin, Stephanie Walker, Araxi Urrutia, Catherine L. Peichel   +5 more
wiley   +1 more source

Magnetic‐Guided Delivery of Antisense Oligonucleotides for Targeted Transduction in Multiple Retinal Explant and Organoid Models

Advanced Science, EarlyView.
Magnetic field‐guided MNP@ATR‐ASO nanocomplexes facilitate the targeted delivery of ASOs to ganglion cells or photoreceptors within retinal explants, thereby enhancing the efficacy of ASOs in correcting pre‐mRNA splicing abnormalities. Furthermore, the nanocomplexes improve ASO penetration and delivery in human retinal and inner ear organoid models ...
Xiuhong Ye, Sihui Chen, Wei Xiong, Fan Wang, Hon Fai Chan, Haocheng Lai, Xiangyu Guo, Tingting Yang, Shuhao Shen, Hang Chen, Wenxuan Wang, Guei‐Sheung Liu, Yonglong Guo, Jiansu Chen   +13 more
wiley   +1 more source

Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model

Frontiers in Pharmacology, 2022
Introduction: Severity and disease progression in people with Cystic Fibrosis (CF) is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides (AO). CFTR
Kelly M. Martinovich, Kelly M. Martinovich, Kelly M. Martinovich, Anthony Kicic, Anthony Kicic, Anthony Kicic, Anthony Kicic, Anthony Kicic, Stephen M. Stick, Stephen M. Stick, Stephen M. Stick, Stephen M. Stick, Russell D. Johnsen, Russell D. Johnsen, Sue Fletcher, Sue Fletcher, Sue Fletcher, Steve D. Wilton, Steve D. Wilton   +18 more
doaj   +1 more source

Universal power law behaviors in genomic sequences and evolutionary models [PDF]

, 2007
We study the length distribution of a particular class of DNA sequences known as 5'UTR exons. These exons belong to the messanger RNA of protein coding genes, but they are not coding (they are located upstream of the coding portion of the mRNA) and are thus less constrained from an evolutionary point of view.
arxiv   +1 more source

Conserved sequence elements associated with exon skipping [PDF]

Nucleic Acids Research, 2003
One of the major forms of alternative splicing, which generates multiple mRNA isoforms differing in the precise combinations of their exon sequences, is exon skipping. While in constitutive splicing all exons are included, in the skipped pattern(s) one or more exons are skipped.
Hanah Margalit, Ruth Sperling, Elana Miriami   +2 more
openaire   +3 more sources

Possibility of exon skipping therapy for Duchenne muscular dystrophy in Russian patients: present and future

Нервно-мышечные болезни
Background. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in children, that occurs between one and three years of age.
E. V. Zinina, M. V. Bulakh, O. P. Ryzhkova, O. A. Shchagina, A. V. Polyakov   +4 more
doaj   +1 more source

The Alternative Choice of Constitutive Exons throughout Evolution [PDF]

PLoS Genet 2007 3(11): e203, 2008
Alternative cassette exons are known to originate from two processes exonization of intronic sequences and exon shuffling. Herein, we suggest an additional mechanism by which constitutively spliced exons become alternative cassette exons during evolution. We compiled a dataset of orthologous exons from human and mouse that are constitutively spliced in
arxiv  

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Molecular Therapy: Methods & Clinical Development, 2023
Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dystrophin protein. Exon duplications represent 6%–11% of mutations, and duplications of exon 2 (Dup2) are
Liubov V. Gushchina, Adrienne J. Bradley, Tatyana A. Vetter, Jacob W. Lay, Natalie L. Rohan, Emma C. Frair, Nicolas Wein, Kevin M. Flanigan   +7 more
doaj  

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
doaj   +1 more source