SMA gene therapy is a one-time infusion that transfers the missing SMN1 gene into the body cells through a virus vector
The gene is inserted into a harmless virus (AAV9) which acts as the vector
This new gene increases survival motor neuron (SMN) protein levels, which improves motor neuron function and increases muscle strength and survival
How does it work on SMA patients?
SMA occurs when there is a missing or abnormal version of the SMN1 gene which is needed to make an essential protein called ‘Survival Motor Neuron’ (SMN) protein.
Lack of SMN protein causes nerves that control muscles (motor neurons) to die. This results in muscles becoming weak and wasting away, with eventual loss of movement, difficulty in breathing and feeding.
Zolgensma works by supplying a fully functioning copy of the SMN1 gene which then helps the body to produce enough SMN protein.