Abstract
Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of congenital alpha-1 antitrypsin deficiency
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have