Transthyretin(TTR) is an transport protein found in liver.It
plays an important role in transporting vitamin A by binding to
Retinol binding protein.
During this process of binding,majority of protein folding
occurs normally but can even lead to few misfolded cases of TTR and
hence can result in an mutated version and accumulation of the
pathologic amlyoid fibrils.
Such a condition is marked as an TTR mediate amyloidosis(ATTR )
which is fatal disease anf can be heriditary in nature.I t can also
include carpel tunel syndrome and cardiomyopathy in later
stages.
RNA interference is an process which will cause an site
direction change in the sequence specific degradation of miRNA.It
will result in inhibition of the protein and thus resulting in no
action.
Therefore, this startegy uses the strategy of silencing the
gene and its expression and reduce the total amount of TTR
production.