Mechanism
LPL (lipoprotein lipase) gene
Assets acting on this target.
- Class
- AAV gene therapy (self-complementary AAV serotype 5 vector carrying a codon-optimized LPL coding sequence under a liver-specific promoter)
- Pathway
- Liver-directed gene replacement restoring lipoprotein lipase activity in primary hypertriglyceridemia/LPL-deficiency patients with recurrent pancreatitis