Publications by authors named "Prajith Stephen"
Pharmacol Rev
December 2023
Article Synopsis
- Systemic diseases of liver origin (SDLO) affect multiple organ systems due to irregular liver function, with examples including primary hyperoxaluria and hereditary transthyretin amyloidosis.
- Nucleic acid therapeutics, particularly antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), are emerging as promising treatments for these conditions, aiming to alter gene expression.
- The article reviews the epidemiology, symptoms, genetic factors, and current treatments of various SDLOs while also discussing the potential benefits and challenges of using nucleic acid therapeutics in liver-targeted therapies.
