Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice
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Muscle stem cells (MuSCs) are the primary source of myogenic progenitors during muscle repair and are essential for the long-term regenerative capacity of skeletal muscle. Following myofiber injury, MuSCs transition from a quiescent to an activated state, ...
The spinal cord is an elongated nervous structure that together with the brain forms the central nervous system. It relays sensory and motor information between the brain and the body, thus controlling most somatic and autonomic body functions. In recent y ...
In spite of decades of research, no feasible method for obtaining sufficient numbers of uncommitted muscle stem cells (MuSCs) for therapy of degenerative muscle diseases exists. One of the most fundamental problems associated with stem cell therapy of musc ...
In order to assess the therapeutic potential of cell-based strategies, it is of paramount importance to elaborate and validate new tools for monitoring the behavior of injected cells in terms of tissue dissemination and engraftment properties. Here, we app ...
The premotor (PM) and primary motor (M1) cortical areas broadcast voluntary motor commands through multiple neuronal pathways, including the corticorubral projection that reaches the red nucleus (RN). However, the respective contribution of M1 and PM to co ...
Spinal cord injury (SCI) disrupts the communication between the brain and the spinal circuits responsible for movement, thereby causing severe motor deficits. Current strategies to restore function to paralyzed limbs have separately investigated electrical ...
In amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, both cell-autonomous and noncell-autonomous mechanisms lead to the selective degeneration of motoneurons (MN). Here, we evaluate the therapeutic potential of gene therapy targeting mutat ...
First human trials involving neuroprosthetic rehabilitation demonstrated recently that significant
functional benefits can be achieved with lumbosacral neuromodulation and reorganized
spared projections. However, complete spinal cord injuries (SCI) wholly ...
Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) are the two most common motoneuron disorders, which share typical pathological hallmarks while remaining genetically distinct. Indeed, SMA is caused by deletions or mutations in the surv ...
Of the seven thousand diseases that are described as rare, 80% of them have an identified genetic cause. With this in the mind, the development of technologies, such as gene therapy, to address the genetic factors involved in these pathologies, might be a ...