Resumo

GONZALEZ-DUARTE, Alejandra. Transthyretin Amyloidosis Therapies: Guiding the Future. Rev. invest. clín. [online]. 2021, vol.73, n.5, pp.310-315.  Epub 18-Out-2021. ISSN 2564-8896.  https://doi.org/10.24875/ric.21000323.

Transthyretin (TTR) amyloidosis (ATTR) is a progressive condition characterized by multiorgan accumulation of amyloid deposits composed of transthyretin (TTR) fibrils. Over the past decades, despite being a rare disease, ATTR amyloidosis has enabled top-tier therapeutics. In the 90s, organ transplantation was the mainstream therapeutic option and fostered distinct approaches, such as combined liver-heart transplant and domino (sequential) liver transplantation. Likewise, several TTR molecule stabilizers were developed successfully. Over the past decade, oriented genetic therapies emerged to prevent, control, and, surprisingly, reverse amyloid deposition. Silencing the TTR gene using different strategies is flourishing, and ongoing trials continue to evaluate diverse approaches to optimize their application. The following perspective describes the currently available treatments for ATTR amyloidosis and the prospects on the potential application of these strategies in other medical fields.

Palavras-chave : Transthyretin amyloidosis; Hereditary amyloidosis; siRNA; Lipid nanoparticles.