•MALAT1
expression and nuclear retention element
(ENE,
which assumes a triple-helical configuration) binding ligands identified
•The
ligands have been further studied for their ability for reverse branching
morphogenesis in tumor-derived organoids
•A
~50% drop in the nuclear MALAT1 copy number demonstrated after treatment with
MALAT1 ENE binding ligands
•Small
molecules targeted to MALAT1 do not recognize a triple helix encoded by a
similar lncRNA,
NEAT1
•Next
step is to apply X-ray crystallography to obtain a
high-resolution structure of MALAT1 in complex with compounds, as a
prerequisite to designing derivatives of these compounds with greater potency
