New Drug Approach that could lead to Cures for wide Range of Diseases

Restoration of testis function in hypogonadotropic hypogonadal mice harboring a misfolded GnRHR mutant by pharmacoperone drug therapy

OHSU researchers develop new drug approach that could lead to cures for wide range of diseases
The ability of drugs called ‘pharmacoperones’ to rescue misfolded proteins and return them to normalcy could someday be an underlying cure to a number of diseases, scientists say

” 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. “

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Oregon Health and Science University, 09 Dec 2013

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