Base editing rescue of spinal muscular atrophy in cells and in mice
Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
Biomarkers and the development of a personalized medicine approach in spinal muscular atrophy
Recent unprecedented advances in treatment for spinal muscular atrophy (SMA) enabled
patients to access the first approved disease modifying therapy for the condition. There are …
patients to access the first approved disease modifying therapy for the condition. There are …
A small-molecule PI3Kα activator for cardioprotection and neuroregeneration
Harnessing the potential beneficial effects of kinase signalling through the generation of
direct kinase activators remains an underexplored area of drug development,,,–. This also …
direct kinase activators remains an underexplored area of drug development,,,–. This also …
Unique Chirality Selection in Neural Cells for D‐Matrix Enabling Specific Manipulation of Cell Behaviors
Y Li, Y Wang, Q Ao, X Li, Z Huang, X Dou… - Advanced …, 2023 - Wiley Online Library
Manipulating neural cell behaviors is a critical issue to various therapies for neurological
diseases and damages, where matrix chirality has long been overlooked despite the proven …
diseases and damages, where matrix chirality has long been overlooked despite the proven …
A scoping review of current and emerging techniques for evaluation of peripheral nerve health, degeneration, and regeneration: Part 1, neurophysiology
Peripheral neuroregeneration research and therapeutic options are expanding
exponentially. With this expansion comes an increasing need to reliably evaluate and …
exponentially. With this expansion comes an increasing need to reliably evaluate and …
[HTML][HTML] Muscle strength and size are associated with motor unit connectivity in aged mice
KA Sheth, CC Iyer, CG Wier, AE Crum, A Bratasz… - Neurobiology of …, 2018 - Elsevier
In older adults, the loss of muscle strength (dynapenia) and the loss of muscle mass
(sarcopenia) are important contributors to the loss of physical function. We sought to …
(sarcopenia) are important contributors to the loss of physical function. We sought to …
High-speed wide-field imaging of microcircuitry using nitrogen vacancies in diamond
JL Webb, L Troise, NW Hansen, LF Frellsen… - Physical Review …, 2022 - APS
The ability to measure the passage of electrical current with high spatial and temporal
resolution is vital for applications ranging from inspection of microscopic electronic circuits to …
resolution is vital for applications ranging from inspection of microscopic electronic circuits to …
[HTML][HTML] Muscle weakness caused by cancer and chemotherapy is associated with loss of motor unit connectivity
Skeletal muscle wasting and weakness caused by cancer and its treatments (known as
“cachexia”) drastically impair quality of life and worsen survival outcomes in cancer patients …
“cachexia”) drastically impair quality of life and worsen survival outcomes in cancer patients …
Modulation of synaptic plasticity, motor unit physiology, and TDP-43 pathology by CHCHD10
Mutations in CHCHD10, a gene coding for a mitochondrial intermembrane space protein,
are associated with Frontotemporal dementia (FTD)-Amyotrophic lateral sclerosis (ALS) …
are associated with Frontotemporal dementia (FTD)-Amyotrophic lateral sclerosis (ALS) …
Selective motor neuron resistance and recovery in a new inducible mouse model of TDP-43 proteinopathy
KJ Spiller, CJ Cheung, CR Restrepo… - Journal of …, 2016 - Soc Neuroscience
Motor neurons (MNs) are the neuronal class that is principally affected in amyotrophic lateral
sclerosis (ALS), but it is widely known that individual motor pools do not succumb to …
sclerosis (ALS), but it is widely known that individual motor pools do not succumb to …