Unraveling the role of GDF5 therapeutic potential in Amyotrophic Lateral Sclerosis
Abstract
Muscle denervation is a common hallmark of several neuromuscular diseases and accounts for skeletal muscle atrophy
and dysfunction associated to their pathophysiology. In non-pathologic muscle, the induction of GDF5/SMAD1/5 pathway is essential for avoiding excessive atrophy but also for promoting re-innervation after nerve damage.
Recently, we demonstrated that alterations of GDF5 pathway can be implicated in human and mouse age-related muscle wasting and that its overexpression prevents muscle mass loss and force decline during ageing in mice.
SMAD1/5 pathway activation has been described as beneficial for motor neuron dysfunction in an Amyotrophic Lateral Sclerosis (ALS) model. We thus hypothesize that GDF5 implementation could have a positive impact on pathophysiology
of the disease. We propose a strategy potentially applicable to different ALS forms and/or to optimize gene therapy-based approaches.
Domains
Life Sciences [q-bio]
Origin : Files produced by the author(s)