Base editing of SMN2 gene restores production of SMN protein, curing spinal muscular atrophy in mice Mar 31, 2023 I suffer from the world's most beautiful disease, and also the most expensive to treat ...

Spinal muscular atrophy (SMA) is a leading cause of infant mortality. Most cases of the disease are caused by mutations in a gene called SMN1 that reduce the production of functional SMN protein, ...

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by mutations in the SMN1 gene that reduce survival motor neuron (SMN) protein expression. Despite progress in the field that ...

Almost all cases of SMA are caused by a mutation in a single gene - SMN1 (survival motor neuron 1) - that reduces the amount of SMN protein inside motor neurons.

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current ...

The most efficient one called SMN 1, that’s what’s missing in spinal muscular atrophy,” Tilton said. Now, the first FDA-approved oral medication to treat SMA, Evrysdi, can turn on the second ...

EXG001-307 is a recombinant AAV (rAAV) gene therapy in Phase 1/2 clinical development for Spinal Muscular Atrophy Type 1 in China. The company plans to file a US IND for EXG001-307 in 2024.

These results imply that using base editing to restore SMN protein levels may help ... correction of SMN2 as a treatment for spinal muscular atrophy ” was ... gene. But SMN1 isn ...

Spinal muscular atrophy (SMA) is a group of serious, progressive diseases that destroys motor neuron cells. SMA treatment aims to reduce symptoms and slow or stop the progression of the disease ...