A married couple, Jean Bennett and Albert Maguire, recently won a $3 million Breakthrough Prize for their groundbreaking work in gene therapy. This award also went to physician Katherine High, recognizing a 25-year journey to develop Luxturna, the first approved gene therapy for a genetic eye disorder called Leber congenital amaurosis (LCA).
Luxturna was approved in the U.S. in 2017 and has changed lives. One patient shared their experience of seeing their child’s face for the first time after treatment. Stories like this highlight the therapy’s profound impact.
Bennett, now retired from the University of Pennsylvania, expressed her joy upon receiving the prize. “It was one of those miraculous eureka moments,” she said. However, she worries about the future of science in the U.S. due to political pressures. She noted that the current administration has undermined scientific research, which could lead to lasting damage.
The couple met during a brain dissection class at Harvard Medical School. At the University of Pennsylvania, they tackled LCA, linked to issues in a gene called RPE65. They persisted despite the challenges. “Being young and naive helped,” Bennett said, referencing her determination.
Their therapy successfully introduced a working version of the faulty gene back into the eye. Along their journey, they adopted two blind dogs named Venus and Mercury, which became part of their family.
Other groundbreaking awards were given for gene therapies addressing sickle cell disease and beta thalassemia. Researchers discovered that disabling a specific gene allowed patients to produce healthier blood cells.
Swee Lay Thein and Stuart Orkin were recognized for this advancement, moving the field rapidly forward. Their work led to Casgevy, a therapy that involves editing patients’ blood stem cells directly. Thein noted, “The field is moving fast,” adding that future treatments may not require lengthy procedures as current methods do.
However, Orkin voiced concerns similar to Bennett’s regarding the barriers facing scientists today. “We were in a golden age of biomedical science,” he lamented, emphasizing the need to protect and promote research.
This couple’s story and others like it remind us of the human side of science. They show how dedication can lead to breakthroughs that improve lives. As scientists confront new challenges, their work remains crucial to our future.
For further insight into gene editing and its applications, you can read more about the advancements in gene-edited crops, which may help tackle health concerns linked to common foods here.
