nL-MAPK8-001

nL-MAPK8-001 is an individualized antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of a specific patient with Neurodevelopmental Disorder with or without Variable Brain Abnormalities (NEDBA) caused by mutations in the *MAPK8IP3* gene. The *MAPK8IP3* gene encodes a scaffold protein (also known as JIP3) that is critical for axonal transport and neuronal development. Mutations in this gene typically result in severe neurodevelopmental delay, intellectual disability, and epilepsy. nL-MAPK8-001 is designed to specifically bind to the patient's mutant mRNA transcripts to modulate protein expression or function, thereby addressing the genetic root cause of the disorder. This therapy is part of n-Lorem's 'n-of-1' program, which develops personalized ASOs for patients with unique, nano-rare genetic conditions.

Other names

MAPK8IP3-targeted antisense oligonucleotideMAPK-8IP3-targeted antisense oligonucleotideMAPK 8IP3-targeted antisense oligonucleotidenL-MAPK8IP3-001nL-MAPK-8IP3-001nL-MAPK 8IP3-001