Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis
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
Antisense oligonucleotides and their applications in rare neurological diseases
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments
Simon McDowall+7 more
doaj +1 more source
Delivery of Antisense Oligonucleotides to the Cornea
Antisense oligonucleotides (ASOs) are synthetic nucleic acids that recognize complementary RNA sequences inside cells and modulate gene expression. In this study, we explore the feasibility of ASO delivery to the cornea. We used quantitative polymerase chain reaction to test the efficacy of a benchmark ASO targeting a noncoding nuclear RNA, Metastasis ...
Xin Gong+8 more
openaire +4 more sources
Identification of candidate regulatory sequences in mammalian 3' UTRs by statistical analysis of oligonucleotide distributions [PDF]
3' untranslated regions (3' UTRs) contain binding sites for many regulatory elements, and in particular for microRNAs (miRNAs). The importance of miRNA-mediated post-transcriptional regulation has become increasingly clear in the last few years. We propose two complementary approaches to the statistical analysis of oligonucleotide frequencies in ...
arxiv
Targeting STAT3 in Cancer with Nucleotide Therapeutics. [PDF]
Signal transducer and activator of transcription 3 (STAT3) plays a critical role in promoting the proliferation and survival of tumor cells. As a ubiquitously-expressed transcription factor, STAT3 has commonly been considered an "undruggable" target for ...
Grandis, Jennifer R+3 more
core +2 more sources
EFTUD2 Regulates Cortical Morphogenesis via Modulation of Caspase‐3 and Aifm1 Splicing Pathways
EFTUD2, a spliceosomal GTPase linked to MFDM, regulates cortical development through apoptotic control. Conditional Eftud2 knockout in murine neural stem cells induces microcephaly and cortical disorganization, while pathogenic variants drive neuronal loss.
Liping Chen+12 more
wiley +1 more source
Specific and non specific hybridization of oligonucleotide probes on microarrays [PDF]
Gene expression analysis by means of microarrays is based on the sequence specific binding of mRNA to DNA oligonucleotide probes and its measurement using fluorescent labels. The binding of RNA fragments involving other sequences than the intended target is problematic because it adds a "chemical background" to the signal, which is not related to the ...
arxiv +1 more source
Under cold stress, MdDSK2a‐like is degraded via the 26S proteasome pathway, thereby alleviating the degradation of MdMTA induced by the 26S proteasome and autophagy pathways mediated by MdDSK2a‐like. The accumulated MdMTA increases the m6A levels of cold‐responsive genes, thereby contributing to increased ROS detoxification, deposition of cell wall, as
Nan Hou+18 more
wiley +1 more source
Parallel Algorithms for DNA Probe Placement on Small Oligonucleotide Arrays [PDF]
Oligonucleotide arrays are used in a wide range of genomic analyses, such as gene expression profiling, comparative genomic hybridization, chromatin immunoprecipitation, SNP detection, etc. During fabrication, the sites of an oligonucleotide array are selectively exposed to light in order to activate oligonucleotides for further synthesis.
arxiv
The Immune Microenvironment: New Therapeutic Implications in Organ Fibrosis
This review summarizes recent advances in understanding the immune microenvironment's role in fibrosis, focusing on phenotypic/functional alterations of immune cells and their dynamic interactions with other cellular constituents within tissues. The authors further explore therapeutic opportunities and challenges in targeting immune microenvironment ...
Xiangqi Chen+6 more
wiley +1 more source