Nano drug delivery systems for antisense oligonucleotides (ASO) therapeutics

T Ramasamy, HB Ruttala, S Munusamy… - Journal of Controlled …, 2022 - Elsevier
Cancer, infectious diseases, and metabolic and hereditary genetic disorders are a global
health burden affecting millions of people, with contemporary treatments offering limited …

[HTML][HTML] Splice-modulating antisense oligonucleotides as therapeutics for inherited metabolic diseases

S Chen, SN Heendeniya, BT Le, K Rahimizadeh… - BioDrugs, 2024 - Springer
Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …

Tip60's novel RNA-binding function modulates alternative splicing of pre-mRNA targets implicated in Alzheimer's disease

A Bhatnagar, K Krick, BC Karisetty… - Journal of …, 2023 - Soc Neuroscience
The severity of Alzheimer9s disease (AD) progression involves a complex interplay of
genetics, age, and environmental factors orchestrated by histone acetyltransferase (HAT) …

[HTML][HTML] Differences in alternative splicing and their potential underlying factors between animals and plants

Y Du, L Cao, S Wang, L Guo, L Tan, H Liu… - Journal of Advanced …, 2023 - Elsevier
Background Alternative splicing (AS), a posttranscriptional process, contributes to the
complexity of transcripts from a limited number of genes in a genome, and AS is considered …

mCherry on Top: A Positive Read-Out Cellular Platform for Screening DMD Exon Skipping Xenopeptide–PMO Conjugates

AL Lessl, J Pöhmerer, Y Lin, U Wilk… - Bioconjugate …, 2023 - ACS Publications
Phosphorodiamidate morpholino oligomers (PMOs) are a special type of antisense
oligonucleotides (ASOs) that can be used as therapeutic modulators of pre-mRNA splicing …

[HTML][HTML] Transcriptome sequencing reveals pathways related to proliferation and differentiation of shitou goose myoblasts

J Chen, S Zhang, G Chen, X Deng, D Zhang, H Wen… - Animals, 2022 - mdpi.com
Simple Summary The Shitou goose, the largest cultivated goose breed in China, has high
research value in meat traits. Muscle development is regulated by genes related to myoblast …

[HTML][HTML] Engineered U1 snRNAs to modulate alternatively spliced exons

ST Hatch, AA Smargon, GW Yeo - Methods, 2022 - Elsevier
Alternative splicing accounts for a considerable portion of transcriptomic diversity, as most
protein-coding genes are spliced into multiple mRNA isoforms. However, errors in splicing …

[HTML][HTML] A Novel Splice Variant of BCAS1 Inhibits β-Arrestin 2 to Promote the Proliferation and Migration of Glioblastoma Cells, and This Effect Was Blocked by …

YH Kuo, HS Hung, CW Tsai, SC Chiu, SP Liu… - Cancers, 2022 - mdpi.com
Simple Summary BCAS1-SV1, a novel splice variant of BCAS1, promotes the proliferation
and migration of glioblastoma cells by directly binding to and inhibiting the tumor suppressor …

Synthesis and Properties of Oligonucleotides Containing LNA-Sulfamate and Sulfamide Backbone Linkages

B Zengin Kurt, D Dhara, AH El-Sagheer… - Organic Letters, 2024 - ACS Publications
Oligonucleotides hold great promise as therapeutic agents but poor bioavailability limits
their utility. Hence, new analogues with improved cell uptake are urgently needed. Here, we …

[HTML][HTML] Therapeutic antisense oligonucleotides in oncology: From bench to bedside

E Çakan, OD Lara, A Szymanowska, E Bayraktar… - Cancers, 2024 - mdpi.com
Simple Summary Antisense oligonucleotide (ASO)-based drugs are a new class of
compounds. In this review, we will discuss ASO technology, including improved delivery …