Twenty-five years ago, the underlying genetic cause for one of the most common and
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …

Spinal muscular atrophy (SMA) is a hereditary neurodegenerative disease with severity
ranging from progressive infantile paralysis and premature death (type I) to limited motor …

Ever since loss of survival motor neuron (SMN) protein was identified as the direct cause of
the childhood inherited neurodegenerative disorder spinal muscular atrophy, significant …

Spinal muscular atrophy (SMA) is an autosomal recessive disorder that is the leading
genetic cause of infantile death. SMA is characterized by loss of motor neurons in the ventral …

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by homozygous
mutations or deletions in the survival of motor neuron (SMN1) gene, encoding the …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by
loss of motor neurons and muscle atrophy, generally presenting in childhood. SMA is …

Objective: Spinal muscular atrophy (SMA) is the number 1 genetic killer of young children. It
is caused by mutation or deletion of the survival motor neuron 1 (SMN1) gene. Although …

Although pigs are used widely as models of human disease, their utility as models has been
enhanced by genetic engineering. Initially, transgenes were added randomly to the genome …

Spinal muscular atrophy (SMA) is a genetic disorder caused by the deletion of the survival
motor neuron 1 (SMN1) gene that leads to loss of motor neurons in the spinal cord. Although …

Spinal muscular atrophy (SMA) is the most common genetically inherited neurodegenerative
disease resulting in infant mortality. SMA is caused by genetic deletion or mutation in the …