ALS, A NEURODEGENERATIVE DISEASE TARGETING MOTOR NEURONS
Amyotrophic Lateral Sclerosis, is a fatal neurological disease targeting lower and upper motor neurons.
People affected by the disease develop at first mild early symptoms including muscle stiffness and twitches. Symptoms usually increase gradually provoking global muscle weakness and muscle wasting.
ALS is classified in three categories, depending on the type
- Classical ALS – involving upper motor neurons in the brain and lower motor neurons in the spinal cord
- PLS – Primary Lateral Sclerosis, involving only upper motor neurons
- PMA – Progressive Muscular Atrophy, involving only lower motor neurons
There is no known cure for ALS a the moment, even though treatments were developed to help alleviate or slow down symptoms of the disease.
It has been shown that mutations of genes associated with ALS like C9ORF72 and SOD1 reduce the survival of motor neurons.
EVALUATE YOUR COMPOUND’S EFFECTS ON ALS MODELS
in vitro models
iPSC-derived motor neurons from ALS patients
- Our cell electrophysiology platform is expert in characterising, validating and performing pharmacological testing on all types of human iPSC-derived neurons.
Thanks to commercially available iPSCs derived from ALS patient donors, we can help you investigate your compound’s activity on human tissue in a condition approaching very closely the physiological condition.
Slice electrophysiology – ALS models
in vivo models
- Our preferred partner Key-Obs offers several predictive models for preclinical in vivo evaluation of treatments for CNS diseases.
Key-Obs offers rodent models exhibiting degeneration of motor neurons and motor deficits similar to those observed in humans.
Please contact us for additional information on our capabilities to help you investigate your compound’s activity on ALS models.
