Whitehead Institute researchers win STAT's Editor's Pick award

Whitehead Institute Member Rudolf Jaenisch and Shawn Liu, a postdoc in the Jaenisch lab, have received the Editor's Pick award for STAT Madness 2019, which pits biomedical research papers from the past year against each other in a "March Madness"-style bracket. Jaenisch and Liu's entry in the competition — a paper published last February in the journal Cell describing how they rescued fragile X neurons using CRISPR/Cas9 — was eliminated in the second round of popular voting. Yet of the 64 entries in the bracket, the STAT staff chose Jaensich and Liu's as their favorite innovation, based on the work's "originality, rigor, and potential impact". 

In the Cell paper, Jaenisch and Liu describe how they restored activity to the fragile X syndrome gene in affected neurons using a modified CRISPR/Cas9 system they developed that removes the methylation — the molecular tags that keep the mutant gene shut off — suggesting that this method may prove to be a useful paradigm for targeting diseases caused by abnormal methylation. Fragile X syndrome is the most frequent cause of intellectual disability in males, affecting 1 out of 3600 boys born.