Metabolic Stability and Targeted Delivery of Oligonucleotides: Advancing RNA Therapeutics Beyond The Liver
P Anand, Y Zhang, S Patil, K Kaur - Journal of Medicinal …, 2025 - ACS Publications
Oligonucleotides have emerged as a formidable new class of nucleic acid therapeutics.
Fully modified oligonucleotides exhibit enhanced metabolic stability and display successful …
Fully modified oligonucleotides exhibit enhanced metabolic stability and display successful …
Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges
R Ruchi, GM Raman, V Kumar… - Expert Opinion on Drug …, 2025 - Taylor & Francis
Introduction Antisense oligonucleotide (ASO) was established as a viable therapeutic option
for genetic disorders. ASOs can target RNAs implicated in various diseases, including …
for genetic disorders. ASOs can target RNAs implicated in various diseases, including …
[HTML][HTML] An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database
J Leckie, A Zia, T Yokota - Genes, 2024 - mdpi.com
Background/Objectives: Antisense oligonucleotide (ASO)-mediated exon-skipping is an
effective approach to restore the disrupted reading frame of the dystrophin gene for the …
effective approach to restore the disrupted reading frame of the dystrophin gene for the …
Recent developments and industry interest in gene therapy for duchenne muscular dystrophy
H Moriyama, T Yokota - Expert Opinion on Biological Therapy, 2024 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease and is known as
the most common genetic muscle disease to manifest in childhood. It is caused by mutations …
the most common genetic muscle disease to manifest in childhood. It is caused by mutations …
[PDF][PDF] Integrating Machine Learning-Based Approaches into the Design of ASO Therapies
J Leckie, T Yokota - 2025 - preprints.org
Rare diseases impose a significant burden on affected individuals, caregivers, and
healthcare systems worldwide. Developing effective therapeutics for these small patient …
healthcare systems worldwide. Developing effective therapeutics for these small patient …