Every March, STAT News, the Boston Globe-sponsored website dedicated to news surrounding health sciences, biotechnology and pharma, hosts its version of a bracket tournament. But instead of basketball, “STAT Madness” crowns the top academic medicine breakthrough of the year.
This year, Johns Hopkins Medicine’s lone STAT Madness entry comes from gastroenterologists Vivek Kumbhari and Florin Selaru, who discovered a way to deliver gene therapy directly to the liver, via the common bile duct. The research has important implications for people who have diseases resulting from a single faulty gene.
Thus far, patients with hereditary monogenic diseases such as hemophilia, cystic fibrosis and Wilson’s disease have seen few benefits from gene therapy, as medicine has lacked a safe and effective way to transport engineered genes to their systems. The gene must be administered, must reach its intended targets, must get into the faulty or damaged cells and then either disrupt or express a protein.
Kumbhari and Selaru, who published their research last summer in the journal Gastrointestinal Endoscopy, used a delicate endoscopic technique to access the liver through the bile ducts. The gene expressed its intended protein, proving the new technique’s effectiveness.
STAT News has pitted their breakthrough against Rice University for the first-round matchup.
Vote for Johns Hopkins in STAT Madness!