[HTML][HTML] Coronavirus RNA proofreading: molecular basis and therapeutic targeting
F Robson, KS Khan, TK Le, C Paris, S Demirbag… - Molecular cell, 2020 - cell.com
Summary The coronavirus disease 2019 (COVID-19) that is wreaking havoc on worldwide
public health and economies has heightened awareness about the lack of effective antiviral …
public health and economies has heightened awareness about the lack of effective antiviral …
[HTML][HTML] Chemistry of peptide-oligonucleotide conjugates: a review
K Klabenkova, A Fokina, D Stetsenko - Molecules, 2021 - mdpi.com
Peptide-oligonucleotide conjugates (POCs) represent one of the increasingly successful
albeit costly approaches to increasing the cellular uptake, tissue delivery, bioavailability …
albeit costly approaches to increasing the cellular uptake, tissue delivery, bioavailability …
Multiple exon skipping in the Duchenne muscular dystrophy hot spots: prospects and challenges
Y Echigoya, KRQ Lim, A Nakamura… - Journal of personalized …, 2018 - mdpi.com
Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly
by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense …
by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense …
Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy
YJ Kim, T Nomakuchi, F Papaleonidopoulou… - Nature …, 2022 - nature.com
Low CFTR mRNA expression due to nonsense-mediated mRNA decay (NMD) is a major
hurdle in developing a therapy for cystic fibrosis (CF) caused by the W1282X mutation in the …
hurdle in developing a therapy for cystic fibrosis (CF) caused by the W1282X mutation in the …
[HTML][HTML] Antisense oligonucleotide therapeutics for cystic fibrosis: recent developments and perspectives
YJ Kim, AR Krainer - Molecules and cells, 2023 - Elsevier
Antisense oligonucleotide (ASO) technology has become an attractive therapeutic modality
for various diseases, including Mendelian disorders. ASOs can modulate the expression of a …
for various diseases, including Mendelian disorders. ASOs can modulate the expression of a …
An overview of recent advances and clinical applications of exon skipping and splice modulation for muscular dystrophy and various genetic diseases
M Rodrigues, T Yokota - Exon Skipping and Inclusion Therapies: Methods …, 2018 - Springer
Exon skipping is a therapeutic approach that is feasible for various genetic diseases and
has been studied and developed for over two decades. This approach uses antisense …
has been studied and developed for over two decades. This approach uses antisense …
An update on diagnostic options and considerations in limb-girdle dystrophies
C Angelini, L Giaretta, R Marozzo - Expert review of …, 2018 - Taylor & Francis
Introduction: Limb-girdle muscular dystrophies (LGMDs) encompass a clinically
heterogeneous group of rare, genetic progressive muscle disorders presenting with …
heterogeneous group of rare, genetic progressive muscle disorders presenting with …
Modified nucleosides, nucleotides and nucleic acids via click azide-alkyne cycloaddition for pharmacological applications
D Perrone, E Marchesi, L Preti, ML Navacchia - Molecules, 2021 - mdpi.com
The click azide= alkyne 1, 3-dipolar cycloaddition (click chemistry) has become the
approach of choice for bioconjugations in medicinal chemistry, providing facile reaction …
approach of choice for bioconjugations in medicinal chemistry, providing facile reaction …
Mesyl phosphoramidate oligonucleotides as potential splice-switching agents: impact of backbone structure on activity and intracellular localization
A series of 2′-deoxy and novel 2′-O-methyl and 2′-O-(2-methoxyethyl)(2′-MOE)
oligonucleotides with internucleotide methanesulfonyl (mesyl, μ) or 1-butanesulfonyl (busyl …
oligonucleotides with internucleotide methanesulfonyl (mesyl, μ) or 1-butanesulfonyl (busyl …
RNA-Based Strategies for Cancer Therapy: In Silico Design and Evaluation of ASOs for Targeted Exon Skipping
Precision medicine in oncology has made significant progress in recent years by approving
drugs that target specific genetic mutations. However, many cancer driver genes remain …
drugs that target specific genetic mutations. However, many cancer driver genes remain …