The Institute for Medical Research

Doctors at Schneider Children’s Medical Center have administered a first-of-its-kind WWOX gene therapy to an 8-month-old baby suffering from WOREE syndrome, a rare and devastating genetic epilepsy. The therapy was originally invented and validated through years of research in the laboratory of Prof. Rami Aqeilan at IMRIC, Faculty of Medicine of Hebrew University of Jerusalem, then further developed for clinical application by Mahzi Therapeutics under the leadership of Dr. Yael Weiss. Led clinically by Dr. Naama Ornstein at Schneider Children’s Medical Center, the treatment involved delivering a functional copy of the missing WWOX gene directly into the child’s brain. The therapy has so far halted the baby’s seizures and represents a major milestone that could pave the way for future gene therapies for rare neurological disorders worldwide.

A new study led by Dr. Yuval Malka of the Faculty of Medicine and Dr. William Feller of the University of Bristol reveals an unexpected mechanism by which cells can produce proteins that promote cancer: “dicing” RNA molecules into short, functional segments, instead of acting according to the standard genetic pattern. The study, published in Cell Reports, undermines conventional perceptions of how genetic information is translated and points to a hitherto unknown mechanism that may influence cancer progression and affect patient response to targeted therapies.