关键词: font-family:Inter, -apple-system, BlinkMacSystemFont, ", font-size:16px, background-color:#FFFFFF, ">转甲状腺素蛋白淀粉样变性、发病机制、沉默剂、稳定剂、基因编辑

Abstract: Amyloidosis represents a category of diseases that stem from the deposition of aberrant proteins, subsequently giving rise to the malfunction of tissues and organs. It has the propensity to trigger multisystemic afflictions, encompassing the peripheral nerves, heart, liver, kidneys, gastrointestinal tract, skin, and eyes. Transthyretin amyloidosis (ATTR) is predominantly associated with anomalies in transthyretin (TTR). TTR is a homotetrameric protein capable of transporting thyroxine and retinol binding protein, and it is principally generated in the liver and the choroid plexus of the brain. ATTR can be subdivided into the variant type (ATTRv) and the wild type (ATTRwt), the principal strategies for treating ATTR involve curtailing the production of TTR in the circulation as well as stabilizing the TTR tetramer structure to preclude its dissociation into monomers. In recent years, a continuous stream of innovative therapies for this ailment has emerged, comprising TTR silencers, TTR stabilizers, and gene editing techniques. This article will meticulously dissect the pathogenesis of ATTR and the latest research advancements of these therapies.

Key words: font-family:Inter, -apple-system, BlinkMacSystemFont, ", font-size:16px, background-color:#FFFFFF, ">transthyretin amyloidosis、pathogenesis、silencing agents、stabilizing agents、gene editing