Down-regulation of NCALD by e.g. siRNA or antisense oligonucleotides (ASOs) can be used to treat SMA and possibly other neurodegenerative diseases like Alzheimer. Potential application solely or as a combined therapy with SMN2 targeted drugs.
On behalf of the University of Cologne, PROvendis offers access to rights for commercial use as well as the opportunity for further co-development.
In vivo studies provided evidence for a therapeutic effectiveness of NCALD down-regulation in SMA mice that express low SMN levels by restoring axon growth. In case of interest, we are pleased to inform you about the current status of the patent.
Riessland et al. (2017) Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis. The American Journal of Human Genetics.
Wirth B, Garbes L, Riessland M. (2013) How genetic modifiers influence the phenotype of spinal muscular atrophy and suggest future therapeutic approaches. Curr. Opin. Genet. Dev. 23(3):330-8.
Ruiz et al. (2010) altered intracellular Ca2+ homeostasis in nerve terminals of severe spinal muscular atrophy in mice. J. Neurosci. 30(3):849-57.