Spinal muscular atrophy: from approved therapies to future therapeutic targets for personalized medicine

H Chaytow, KME Faller, YT Huang… - Cell Reports Medicine, 2021 - cell.com
Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the
most severe cases and when left untreated, leads to death within the first two years of life …

Current status of microRNA-based therapeutic approaches in neurodegenerative disorders

S Paul, LA Bravo Vázquez, S Pérez Uribe… - Cells, 2020 - mdpi.com
MicroRNAs (miRNAs) are a key gene regulator and play essential roles in several biological
and pathological mechanisms in the human system. In recent years, plenty of miRNAs have …

[HTML][HTML] Serum miRNAs miR-206, 143-3p and 374b-5p as potential biomarkers for amyotrophic lateral sclerosis (ALS)

R Waller, EF Goodall, M Milo, J Cooper-Knock… - Neurobiology of …, 2017 - Elsevier
Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative condition characterized by
loss of motor neurones and progressive muscle wasting. There is no diagnostic test for ALS …

Regulation of class IIa HDAC activities: it is not only matter of subcellular localization

E Di Giorgio, C Brancolini - Epigenomics, 2016 - Taylor & Francis
In response to environmental cues, enzymes that influence the functions of proteins, through
reversible post-translational modifications supervise the coordination of cell behavior like …

HDAC4 in cancer: A multitasking platform to drive not only epigenetic modifications

E Cuttini, C Goi, E Pellarin, R Vida… - Frontiers in molecular …, 2023 - frontiersin.org
Controlling access to genomic information and maintaining its stability are key aspects of
cell life. Histone acetylation is a reversible epigenetic modification that allows access to DNA …

Role of MicroRNAs, aptamers in neuroinflammation and neurodegenerative disorders

I Khan, K Preeti, V Fernandes, DK Khatri… - Cellular and molecular …, 2022 - Springer
Exploring the microRNAs and aptamers for their therapeutic role as biological drugs has
expanded the horizon of its applicability against various human diseases, explicitly targeting …

miRNAs as biomarkers of neurodegenerative disorders

V Viswambharan, I Thanseem, MM Vasu… - Biomarkers in …, 2017 - Taylor & Francis
Neurodegenerative diseases (NDDs) are the result of progressive deterioration of neurons,
ultimately leading to disabilities. There is no effective cure for NDDs at present; ongoing …

mi RNA in spinal muscular atrophy pathogenesis and therapy

F Magri, F Vanoli, S Corti - Journal of cellular and molecular …, 2018 - Wiley Online Library
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease
characterized by the selective death of lower motor neurons in the brain stem and spinal …

Drug screening and drug repositioning as promising therapeutic approaches for spinal muscular atrophy treatment

G Menduti, DM Rasà, S Stanga, M Boido - Frontiers in pharmacology, 2020 - frontiersin.org
Spinal muscular atrophy (SMA) is the most common genetic disease affecting infants and
young adults. Due to mutation/deletion of the survival motor neuron (SMN) gene, SMA is …

Micro-RNAs in ALS muscle: Differences in gender, age at onset and disease duration

V Pegoraro, A Merico, C Angelini - Journal of the Neurological Sciences, 2017 - Elsevier
Few studies have explored the role of microRNAs (or miRNAs) in Amyotrophic Lateral
Sclerosis (ALS) muscle, possibly because of the difficulty in obtaining samples and because …