Understanding human gene function in living organisms has long been hampered by fundamental differences between species.

The first patient has been dosed in a Phase 2 clinical trial testing RAG-17 in people with ALS who carry mutations in the ...

A new CRISPR-based approach for tuning gene expression up and down allowed identification of how other genes' expression responds to changes of transcription factors.

UMass Chan Medical School scientists have nearly tripled the known number of potential regulatory elements in the genome to 2 ...

Victoria Gray spent 34 years battling the debilitating pain of sickle cell disease. Then she volunteered to be the world's first "prototype" for a CRISPR therapy, based on technology invented at UC ...

University of Navarra (Spain) researchers have developed RNACOREX, a new open-source software capable of identifying gene regulation networks with applications in cancer survival analysis. The tool, ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

PHILADELPHIA (WPVI) -- A miracle baby who was treated with a pioneering gene-therapy at Children's Hospital of Philadelphia has reached a major milestone as he took his first steps. KJ Muldoon was the ...

With a new study in the journal Cell, researchers at Stanford University and Stockholm University have contributed to increased knowledge about gene regulation in human cells. How genes are turned on ...

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...