mi<scp>RNA</scp> in spinal muscular atrophy pathogenesis and therapy

Magri, Francesca; Vanoli, Fiammetta; Corti, Stefania

doi:10.1111/jcmm.13450

Cited by 50 publications

(44 citation statements)

References 76 publications

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“…[27][28][29] MiRNAs were considered as an oncogene or cancer suppressor gene in multiple cancers via interfering with transcription or inhibit translation. [27][28][29] MiRNAs were considered as an oncogene or cancer suppressor gene in multiple cancers via interfering with transcription or inhibit translation.…”

Section: Discussionmentioning

confidence: 99%

MiR‐216b inhibits osteosarcoma cell proliferation, migration, and invasion by targeting Forkhead Box M1

Wang

Guo

et al. 2018

J of Cellular Biochemistry

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Osteosarcoma (OS) is considered the most common type of primary malignant bone tumor, which has a high rate of mortality in children and adolescents. However, the current treatment methods for OS are ineffective. Therefore, there is an urgent requirement to identify the critical targets. This study aimed to identify the roles and significance of microRNA‐216b (miR‐216b) in OS. To explore the cellular and molecular functions of miR‐216b and Forkhead Box M1 (FoxM1) in OS, the expression of miR‐216b and FoxM1 at the transcriptional level was measured using quantitative real‐time PCR (qRT‐PCR). Wound healing assay, 3‐[4,5‐dimethylthiazol‐2‐yl]‐2,5 diphenyltetrazolium bromide assay (MTT) assay, flow cytometry, and transwell invasion assay were conducted to study the function of miR‐216b and FoxM1 in OS cells. Dual luciferase reporter assay was performed to identify the relationships between miR‐216b and FoxM1. qRT‐PCR results revealed that miR‐216b expression was significantly downregulated, and FoxM1 was observed to be significantly upregulated in human OS cell lines (MG‐63) and tissues. MTT data showed that upregulation of miR‐216b expression led to cell growth inhibition in MG‐63 cells. The results of the invasion assay and wound healing assay illustrated that miR‐216b upregulation or FoxM1 downregulation could inhibit the invasion and migration in MG‐63 cells. In vivo, the tumor volume was significantly decreased by miR‐194 mimic treatment compared with the control group. Furthermore, the results of the luciferase assay indicated that FoxM1 is a direct target of miR‐216b. These findings may provide novel insights into the molecular mechanism of miR‐216b and FoxM1 in the progression of OS, and suggested that miR‐216b may serve as a potential tumor inhibitor of OS by targeting FoxM1.

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Section: Discussionmentioning

confidence: 99%

MiR‐216b inhibits osteosarcoma cell proliferation, migration, and invasion by targeting Forkhead Box M1

Wang

Guo

et al. 2018

J of Cellular Biochemistry

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“…These factors are not sufficient to explain the wide variation from patient to patient in outcomes upon treatment. Biomarkers such as microRNA or full‐length SMN RNA could help to identify those patients most likely to be clinical responders . To correlate the clinical response at 2 months or 6 months with milestone acquisition later on could also help to identify patients for whom treatment should be continued or not.…”

Section: Existing Gapsmentioning

confidence: 99%

Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Gidaro

Servais

2018

Develop Med Child Neuro

129

111

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Spinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival motor neuron 1 gene (SMN1); it affects 1 in 11 000 newborn infants. The most severe and most common form, type 1 SMA, is associated with early mortality in most cases and severe disability in survivors. Nusinersen, an antisense oligonucleotide, promotes production of full-length protein from the pseudogene SMN2. Nusinersen treatment prolongs survival of patients with type 1 SMA and allows motor milestone acquisition. Patients with type 2 SMA also show progress on different motor scales after nusinersen treatment. Nusinersen was recently approved by the European Medicines Agency and the US Food and Drug Administration; it is now reimbursed in several European countries and in the USA. In Australia, the transition from expanded access programme to commercial availability is coming soon. In New Zealand, an expanded access programme is opened, and in Canada price negotiation for the treatment is in progress. In this review we exemplify the clinical benefit of nusinersen in subgroups of patients with SMA. Nusinersen represents the first efficacious marked approved drug in type 1 and type 2 SMA. Different knowledge gaps, such as results in older patients, in patients with permanent ventilation, in patients with neonatal forms, or in patients after spinal fusion, still need to be addressed. WHAT THIS PAPER ADDS: Identifies gaps in knowledge about the efficacy of nusinersen in broader populations of patients with spinal muscular atrophy. Identifies open questions in populations of patients where proof of efficacy is available.

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“…In a SMA mice model, a correlation was found between motor neuron degeneration and down-regulation of miR-9, which targets heavy neurofilament subunit [ 121 ]. Since then, plenty of miRNAs have been acknowledged to have a role in SMA development, including miR-132, miR -206, miR -183, miR -335-5p, miR -431, miR -375, miR -2, mirR-218, miR-335-5p and miR-100-5p [ 122 , 123 , 124 ]. At least miR-9, miR-206, miR-183, and miR-375 have been pointed out to hold the potential to be explored as therapeutic targets for SMA [ 122 ].…”

Section: Spinal Muscular Atrophy (Sma)mentioning

confidence: 99%

Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

Paul

Bravo-Vázquez

Uribe

et al. 2020

Cells

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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 been identified to be involved in the development of neurodegenerative disorders (NDDs), thus making them an attractive option for therapeutic approaches. Hence, in this review, we provide an overview of the current research of miRNA-based therapeutics for a selected set of NDDs, either for their high prevalence or lethality, such as Alzheimer’s, Parkinson’s, Huntington’s, Amyotrophic Lateral Sclerosis, Friedreich’s Ataxia, Spinal Muscular Atrophy, and Frontotemporal Dementia. We also discuss the relevant delivery techniques, pertinent outcomes, their limitations, and their potential to become a new generation of human therapeutic drugs in the near future.

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miRNA in spinal muscular atrophy pathogenesis and therapy

Cited by 50 publications

References 76 publications

MiR‐216b inhibits osteosarcoma cell proliferation, migration, and invasion by targeting Forkhead Box M1

MiR‐216b inhibits osteosarcoma cell proliferation, migration, and invasion by targeting Forkhead Box M1

Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

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