ABSTRACT RNA interference (RNAi) provides researchers with a versatile means to
modulate target gene expression. The major forms of RNAi molecules, genome-derived …

Compared with traditional drug therapy, nanomedicines exhibit intriguing biological features
to increase therapeutic efficiency, reduce toxicity and achieve targeting delivery. This review …

The systemic amyloidoses are a broad spectrum of diseases that result from misfolding of
proteins that aggregate into b-sheet amyloid fibrils. Over 35 amyloidogenic precursor …

Background Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening
disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein …

Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into
the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral …

The recent approval of three drugs for the treatment of amyloid transthyretin (ATTR)
amyloidosis, both hereditary and wild-type, has opened a new era in the care of these …

Nucleic acid-based therapies have become the third major drug class after small molecules
and antibodies. The role of nucleic acid-based therapies has been strengthened by recent …

RNA has sparked a revolution in modern medicine, with the potential to transform the way
we treat diseases. Recent regulatory approvals, hundreds of new clinical trials, the …

RNA molecules have emerged as promising clinical therapeutics due to their ability to
target" undruggable" proteins or molecules with high precision and minimal side effects …

Aim Systematic evidence on the prevalence and clinical outcome of transthyretin
amyloidosis (ATTR) is missing. We explored:(i) the prevalence of cardiac amyloidosis in …