Transthyretin amyloidosis ATTR

Familial transthyretin amyloidosis (ATTR) is a hereditary degenerative disease which is closely associated with single amino acid substitutions in the plasma protein transthyretin (TTR), a 127 amino acid protein (Mr 13,761 Da) that is tetrameric in its native state.28 The clinical manifestations of ATTR are related to specific mutations of TTR (e.g., Val30 - Met and Thr60 — Ala). The definitive diagnosis is often established on the basis of the... 

Familial amyloidoses are rare autosomal dominant disorders encompassing more than seven different genes and protein products. Transthyretin amyloidosis (ATTR) is the most common hereditary form of the disease, newly affecting... 

VEGF) in patients with advanced solid tumors with liver involvanent (Tabemero et al 2013). Using the DLin-DMA, Alnylam Pharmaceuticals Inc. developed another SNALP encapsulating a siRNA targeting transthyretin (ALN-TTROl) for the treatment of transthyretin mediated amyloidosis (ATTR) that in 2012 completed a phase 1 clinical trial (Table 14.1). 

Bergstrom J, Murphy C, Eulitz M, Weiss DT, Westermark GT, Solomon A, Westermark P (2001) Codeposition of apolipoprotein A-IV and transthyretin in senile systemic (ATTR) amyloidosis. Biochem Biephys Res Commun 285 903—908... 

ATTR, transthyretin mediated amyloidosis HAI, hepatic arterial infusion LDL-C, LDL-cholesterol METAVIR F3-4, moderate to extensive hepatic fibrosis JTR, transthyretin. 

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