Celena Lozano's son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome.

Earlier this year, when headlines announced that a child with a different rare disease, known as Baby KJ, had been treated using CRISPR in a major breakthrough, Celena, a neuroscience Ph.D. candidate, found that many in the PURA community had major questions, with comments like “This happened in just six months. When will this be available for our PURA kids?” appearing on Facebook groups.

Celena and STAT reporter Jason Mast joined the podcast to discuss why CRISPR for PURA syndrome is particularly difficult, what it’s like to be both a rare disease parent and a scientist, and how crucial parents are to research.