A team led by a longtime Oregon Health & Science University
researcher has demonstrated in mice what could be a revolutionary new
technique to cure a wide range of human diseases — from cystic fibrosis
to cataracts to Alzheimer's disease — that are caused by "misfolded"
protein molecules.
Misfolded protein molecules, caused by gene mutation, are capable of
maintaining their function but are misrouted within the cell and can’t
work normally, thus causing disease. The OHSU team discovered a way to
use small molecules that enter cells, fix the misfolded proteins and
allow the proteins to move to the correct place and function normally
again.
Credit: http://www.ohsu.edu
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