[HTML][HTML] RNA and disease
Cellular functions depend on numerous protein-coding and noncoding RNAs and the RNA-
binding proteins associated with them, which form ribonucleoprotein complexes (RNPs) …
binding proteins associated with them, which form ribonucleoprotein complexes (RNPs) …
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …
[HTML][HTML] A long noncoding RNA controls muscle differentiation by functioning as a competing endogenous RNA
Recently, a new regulatory circuitry has been identified in which RNAs can crosstalk with
each other by competing for shared microRNAs. Such competing endogenous RNAs …
each other by competing for shared microRNAs. Such competing endogenous RNAs …
Theoretic applicability of antisense‐mediated exon skip** for Duchenne muscular dystrophy mutations
Antisense‐mediated exon skip** aiming for reading frame restoration is currently a
promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach …
promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach …
Molecular and cellular mechanisms of skeletal muscle atrophy: an update
A Fanzani, VM Conraads, F Penna… - Journal of cachexia …, 2012 - Springer
Skeletal muscle atrophy is defined as a decrease in muscle mass and it occurs when protein
degradation exceeds protein synthesis. Potential triggers of muscle wasting are long-term …
degradation exceeds protein synthesis. Potential triggers of muscle wasting are long-term …
Molecular mechanisms of muscular dystrophies: old and new players
The study of the muscle cell in the muscular dystrophies (MDs) has shown that mutant
proteins result in perturbations of many cellular components. MDs have been associated …
proteins result in perturbations of many cellular components. MDs have been associated …
Delivery is key: lessons learnt from develo** splice‐switching antisense therapies
The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐
clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety …
clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety …
miRNAs as serum biomarkers for Duchenne muscular dystrophy
Dystrophin absence in Duchenne muscular dystrophy (DMD) causes severe muscle
degeneration. We describe that, as consequence of fibre damage, specific muscle‐miRNAs …
degeneration. We describe that, as consequence of fibre damage, specific muscle‐miRNAs …
Genetic therapies for RNA mis-splicing diseases
SM Hammond, MJA Wood - Trends in genetics, 2011 - cell.com
RNA mis-splicing diseases account for up to 15% of all inherited diseases, ranging from
neurological to myogenic and metabolic disorders. With greatly increased genomic …
neurological to myogenic and metabolic disorders. With greatly increased genomic …
Exon-skip** advances for Duchenne muscular dystrophy
L Echevarría, P Aupy… - Human molecular genetics, 2018 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a fatal genetic disorder characterized by
progressive muscle wasting that has currently no cure. Exon-skip** strategy represents …
progressive muscle wasting that has currently no cure. Exon-skip** strategy represents …