Spinal muscular atrophy (SMA) is a lower motor neuron disease with autosomal recessive
inheritance. The first cases of SMA were reported by Werdnig in 1891. Although the …

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurological disease affecting
both upper and lower motor neurons. In the United States alone, there are 16,000–20,000 …

Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Gene therapy is defined as the administration of genetic material to modify, manipulate gene
expression or alter the properties of living cells for therapeutic purposes. Recent advances …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by
mutations in the SMN1 gene. Despite the development of various therapies, outcomes can …

Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic
approaches have been developed to correct aberrant alternative splicing events for the …

Neurological and neuropsychiatric disorders are the main risks for the health care system,
exhibiting a huge socioeconomic load. The available range of pharmacotherapeutics mostly …

The development of new possible treatments for C9orf72-related ALS and the possibility of
early identification of subjects genetically at risk of developing the disease is creating a …

Nucleic acid therapeutics have been attracting attention as novel drug discovery modalities
for intractable diseases, including amyotrophic lateral sclerosis. This review provides an …

Amyotrophic lateral sclerosis (ALS) is a prototypical neurodegenerative disease
characterized by progressive degeneration of motor neurons to severely effect the …