Fixing nature’s genetic mistakes in the womb

Tippi MacKenzie, MD, UCSF-CTSI Catalyst Program Awardee, was recently featured in an in-depth news story about her team's pioneering approaches in fetal gene therapy.

By the time a baby is born with a catastrophic genetic disease, medical help is too little, too late.

UC San Francisco doctors are studying a bolder and better remedy, far earlier: repairing genes before birth, stopping disease before it starts.

Experts recently met on campus to plan how to best undertake this pioneering approach, called fetal gene therapy, as well as its potential pitfalls. Strategies will be discussed again at a Wednesday gathering.

“It would be a first in humans,” and a collaboration between leaders at a half dozen university medical centers, said Dr. Tippi MacKenzie, a pediatric and fetal surgeon who is leading the effort – still two to five years away — at UCSF’s Benioff Children’s Hospital.  “That’s one of the reasons we’re treading so carefully.”

“Treating the fetus could prevent illness and sustain a pregnancy,” she said.