News and insights

Subscribe to our newsletter

A commonly inherited gene deletion can increase the likelihood of immune complications following bone marrow transplantation, an international team of researchers reports in the November 22 advance online issue of Nature Genetics. When the gene, called UGT2B17, is missing from the donor's genome but present in the recipient's, transplants have a significantly greater risk of a serious side-effect known as graft-versus-host disease, in which immune cells from the donor attack tissues in the recipient.

 

Scientists have devised an innovative way to disarm a key protein considered to be "undruggable," meaning that all previous efforts to develop a drug against it have failed. Their discovery, published in the November 12 issue of Nature, lays the foundation for a new kind of therapy aimed directly at a critical human protein - one of a few thousand so-called transcription factors - that could someday be used to treat a variety of diseases, especially multiple types of cancer.

 

Discovering drugs is a lot like target practice. While the intended targets — specific molecules in the human body, usually proteins — are far smaller than the typical bull's eye, it can be said that hitting some seems much more difficult than hitting others.

In scientific terms, these tricky targets — molecules for which drugs have been attempted but ultimately failed, or which have certain properties that make them too challenging to even consider — have earned an influential label: Undruggable.