Circulating microRNAs as potential biomarkers and therapeutic targets in spinal muscular atrophy

TH Chen - Therapeutic advances in neurological disorders, 2020 - journals.sagepub.com
Spinal muscular atrophy (SMA), a leading genetic cause of infant death, is a
neurodegenerative disease characterized by the selective loss of particular groups of motor …

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 …

Cell-type-specific miR-431 dysregulation in a motor neuron model of spinal muscular atrophy

MH Wertz, K Winden, P Neveu, SY Ng… - Human molecular …, 2016 - academic.oup.com
Spinal muscular atrophy (SMA) is an autosomal-recessive pediatric neurodegenerative
disease characterized by selective loss of spinal motor neurons. It is caused by mutation in …

[HTML][HTML] Altered levels of MicroRNA-9,-206, and-132 in spinal muscular atrophy and their response to antisense oligonucleotide therapy

F Catapano, I Zaharieva, M Scoto, E Marrosu… - … Therapy-Nucleic Acids, 2016 - cell.com
The identification of noninvasive biomarkers to monitor the disease progression in spinal
muscular atrophy (SMA) is becoming increasingly important. MicroRNAs (miRNAs) regulate …

[HTML][HTML] Circulating myomiRs as potential biomarkers to monitor response to nusinersen in pediatric SMA patients

S Bonanno, S Marcuzzo, C Malacarne, E Giagnorio… - Biomedicines, 2020 - mdpi.com
Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by mutations in
survival motor neuron (SMN) 1 gene, resulting in a truncated SMN protein responsible for …

[HTML][HTML] Multifaceted roles of microRNAs: From motor neuron generation in embryos to degeneration in spinal muscular atrophy

TH Chen, JA Chen - Elife, 2019 - elifesciences.org
Two crucial questions in neuroscience are how neurons establish individual identity in the
developing nervous system and why only specific neuron subtypes are vulnerable to …

[HTML][HTML] Identification of novel CSF-derived miRNAs in treated paediatric onset spinal muscular atrophy: an exploratory study

AM D'Silva, D Kariyawasam, P Venkat, C Mayoh… - Pharmaceutics, 2023 - mdpi.com
The availability of disease modifying therapies for spinal muscular atrophy (SMA) have
created an urgent need to identify clinically meaningful biomarkers that provide insight into …

The potential role of miRNA therapies in spinal muscle atrophy

G Gandhi, S Abdullah, AI Foead, WWY Yeo - Journal of the Neurological …, 2021 - Elsevier
Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of full-
length survival motor neuron (SMN) protein due to the loss of the survival motor neuron 1 …

Response of plasma microRNAs to nusinersen treatment in patients with SMA

IT Zaharieva, M Scoto… - Annals of clinical …, 2022 - Wiley Online Library
Objective Spinal muscular atrophy (SMA) is a common genetic cause of infant mortality.
Nusinersen treatment ameliorates the clinical outcome of SMA, however, some patients …

SMN regulates axonal local translation via miR-183/mTOR pathway

MJ Kye, ED Niederst, MH Wertz… - Human molecular …, 2014 - academic.oup.com
Reduced expression of SMN protein causes spinal muscular atrophy (SMA), a
neurodegenerative disorder leading to motor neuron dysfunction and loss. However, the …