Spinal muscular atrophy is a destructive motor neuron disease that mostly affects children and represents the most common cause of hereditary infant mortality. It is mainly caused by harmful variants in survival of motor neuron (SMN1), which drive to depletion of the survival motor neuron protein (SMN), along with specific immune organ defects that compromise immune function and consequently the survival and quality of life of SMA patients. In this work, we present an application of a computational modeling approach in order to analyze and suggest possible therapeutic interventions through the inhibitory strategies directed against spinal muscular atrophy.
Computational modeling approach to suggest possible therapeutic interventions in spinal muscular atrophy
Russo G.;
2019-01-01
Abstract
Spinal muscular atrophy is a destructive motor neuron disease that mostly affects children and represents the most common cause of hereditary infant mortality. It is mainly caused by harmful variants in survival of motor neuron (SMN1), which drive to depletion of the survival motor neuron protein (SMN), along with specific immune organ defects that compromise immune function and consequently the survival and quality of life of SMA patients. In this work, we present an application of a computational modeling approach in order to analyze and suggest possible therapeutic interventions through the inhibitory strategies directed against spinal muscular atrophy.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.