Publications by authors named "Hae-Won Son"
The endocannabinoid system (ECS) is widely studied due to its interactions with cannabis and its role in modulating physiological responses. While most research has focused on the effects of cannabis on adult ECSs, recent studies have begun to investigate the role of the ECS in developing organisms. However, little is known about the spatial or temporal expression of these receptors during early development.
View Article and Find Full Text PDFRecent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy.
Methods Mol Biol
April 2019
Article Synopsis
- Spinal muscular atrophy (SMA) is a genetic disorder caused by mutations in the SMN1 gene, leading to inadequate SMN protein production and resulting in motor neuron loss, muscle weakness, and paralysis.
- The related gene SMN2 produces mostly non-functional proteins due to splicing issues, but targeting the intronic splicing silencer (ISS-N1) with antisense oligonucleotides can promote the inclusion of necessary exon 7, potentially restoring SMN levels.
- Nusinersen (Spinraza) is the first FDA-approved treatment that employs this strategy, and ongoing research explores new molecular tools to enhance treatment effectiveness and improve patient outcomes.