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 …

The role of survival motor neuron protein (SMN) in protein homeostasis

H Chaytow, YT Huang, TH Gillingwater… - Cellular and Molecular …, 2018 - Springer
Ever since loss of survival motor neuron (SMN) protein was identified as the direct cause of
the childhood inherited neurodegenerative disorder spinal muscular atrophy, significant …

Autophagy in neurodegeneration: New insights underpinning therapy for neurological diseases

O Corti, K Blomgren, A Poletti… - Journal of …, 2020 - Wiley Online Library
In autophagy long‐lived proteins, protein aggregates or damaged organelles are engulfed
by vesicles called autophagosomes prior to lysosomal degradation. Autophagy dysfunction …

[HTML][HTML] Autophagy in sarcopenia: Possible mechanisms and novel therapies

G Xie, H Jin, H Mikhail, V Pavel, G Yang, B Ji… - Biomedicine & …, 2023 - Elsevier
With global population aging, age-related diseases, especially sarcopenia, have attracted
much attention in recent years. Characterized by low muscle strength, low muscle quantity or …

Isoquercitrin delays denervated soleus muscle atrophy by inhibiting oxidative stress and inflammation

Y Shen, Q Zhang, Z Huang, J Zhu, J Qiu, W Ma… - Frontiers in …, 2020 - frontiersin.org
Although denervated muscle atrophy is common, the underlying molecular mechanism
remains unelucidated. We have previously found that oxidative stress and inflammatory …

Mitochondrial dysfunction in spinal muscular atrophy

E Zilio, V Piano, B Wirth - International Journal of Molecular Sciences, 2022 - mdpi.com
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by
recessive mutations in the SMN1 gene, globally affecting~ 8–14 newborns per 100,000. The …

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 …

Stasimon contributes to the loss of sensory synapses and motor neuron death in a mouse model of spinal muscular atrophy

CM Simon, M Van Alstyne, F Lotti, E Bianchetti… - Cell reports, 2019 - cell.com
Reduced expression of the survival motor neuron (SMN) protein causes the
neurodegenerative disease spinal muscular atrophy (SMA). Here, we show that adeno …

Revisiting the role of mitochondria in spinal muscular atrophy

R James, H Chaytow, LM Ledahawsky… - Cellular and Molecular …, 2021 - Springer
Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease of variable
clinical severity that is caused by mutations in the survival motor neuron 1 (SMN1) gene …

Lipid microcapsules promoted neural stem cell survival in the infarcted area of mice with ischemic stroke by inducing autophagy

R Xu, C Duan, Z Meng, J Zhao, Q He… - ACS Biomaterials …, 2022 - ACS Publications
Intracerebral transplantation of neural stem cells (NSCs) for ischemic stroke treatment has
been demonstrated to be inefficient, with only< 5% of delivered cells being retained …