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 …

All muscle movements, including breathing, walking, and fine motor skills rely on the
function of the spinal motor neuron to transmit signals from the brain to individual muscle …

Abstract Spinal Muscular Atrophy (SMA) is caused by genetic mutations in the SMN1 gene,
resulting in drastically reduced levels of Survival of Motor Neuron (SMN) protein. Although …

Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and spinal
muscular atrophy (SMA) are the most prevalent neuromuscular disorders (NMDs) in children …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, predominantly
affects high metabolic tissues including motor neurons, skeletal muscles and the heart …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by
recessive mutations in the SMN1 gene, globally affecting~ 8–14 newborns per 100,000. The …

Human neuronal models of hereditary spastic paraplegias (HSP) that recapitulate disease-
specific axonal pathology hold the key to understanding why certain axons degenerate in …

We report a method to generate a 3D motor neuron model with segregated and directed
axonal outgrowth. iPSC-derived motor neurons are cultured in extracellular matrix gel in a …

The approved gene therapies for spinal muscular atrophy (SMA), caused by loss of survival
motor neuron 1 (SMN1), greatly ameliorate SMA natural history but are not curative. These …

Differentiation of human pluripotent stem cells (hPSCs) in vitro offers a way to study cell
types that are not accessible in living patients. Previous research suggests that hPSCs …