[Music]
while RNA editing is critical for
generating the hundreds of thousands of
proteins that Keep Us Alive splicing
errors can lead to devastating health
problems one example is an often fatal
disease called spinal muscular atrophy
or
SMA it's the number one genetic cause of
infant
death SMA is caused by too little of a
vital protein called survival of motor
neuron or SMN in the spinal cords motor
neurons too little of this protein
causes the motor neurons to die and the
muscles they control to waste
away the smn1 gene which produces The
SMN protein is missing or damaged Beyond
repair in SMA patients humans have a
second SMN producing Gene called
smn2 but this Gene is a poor backup
because it produces very little
functional SMN protein