Genetically Engineered Skin Grafts Offer Hope for Epidermolysis Bullosa Patients

In a groundbreaking advancement for dermatological treatments, genetically engineered skin grafts derived from patients' own cells have been shown to heal chronic wounds in individuals suffering from severe dystrophic epidermolysis bullosa (EB). This innovative approach, revealed in a phase 3 clinical trial led by Stanford Medicine, holds promise for enhancing the quality of life for patients afflicted by this debilitating genetic condition.

Epidermolysis bullosa is characterized by extremely fragile skin that blisters and wounds easily, typically resulting from mutations affecting collagen VII, a crucial protein that provides structural integrity to the skin layers. The recent clinical trial, published in The Lancet on June 23, 2025, involved 11 patients and demonstrated that those treated with genetically engineered grafts experienced significantly better healing outcomes compared to those receiving standard care.

"With our novel gene therapy technique, we successfully treated the hardest-to-heal wounds, which were usually also the most painful ones for these patients," stated Dr. Jean Tang, MD, PhD, the study's lead author and a professor of dermatology at Lucile Packard Children’s Hospital Stanford. The grafts, which received U.S. Food and Drug Administration (FDA) approval on April 29, represent a culmination of over two decades of research conducted at Stanford.

The trial participants reported a marked reduction in pain and itching associated with their wounds. For instance, Charlotte Brown, a 20-year-old participant from Birmingham, Alabama, noted that the grafts significantly alleviated her pain, enabling her to enjoy a more active and fulfilling lifestyle, including holding a job she loves.

"It’s honestly life-changing," Brown remarked. "I feel so much better." The study involved a unique design where pairs of wounds on the same patient were compared: one treated with the graft and the other with conventional methods. Results showed that at 24 weeks post-treatment, 81% of grafted wounds were at least half healed, compared to only 16% in the control group.

Stanford Medicine's pioneering work in this field dates back to the early 2000s when researchers, including Dr. Paul Khavari and Dr. Zurab Siprashvili, first developed a method to engineer skin cells by introducing a corrected version of the collagen VII gene. This foundational research paved the way for the creation of personalized skin grafts that are tailored to each patient’s unique genetic makeup, thus minimizing the risk of immune rejection.

The treatment process begins with a small biopsy from the patient’s uninjured skin, which is then sent to a laboratory where the skin cells are genetically modified. After approximately 25 days, the cells are transformed into skin grafts that can be sutured onto the affected areas.

In addition to the immediate healing benefits, the long-term implications of this treatment could be significant in reducing the risk of skin cancer and other complications associated with chronic wounds in EB patients. The research team plans to follow the trial participants for an extended period of up to 15 years to monitor the grafts' efficacy and safety.

The development of these grafts aligns with a broader effort to improve treatment options for EB, which affects approximately 1 in 500,000 individuals. The disease is often referred to as a 'butterfly skin' condition due to the fragility of the skin, which is reminiscent of a butterfly's wings. Patients experience not only physical pain but also psychological burdens due to their condition.

Dr. Tang expressed optimism for the future, stating, "Who would have thought that an experiment in a Stanford lab would lead to a personalized therapy for EB patients? Now there’s a lot of hope." As this innovative treatment becomes available at select hospitals across the United States, it represents a significant leap forward in the management of epidermolysis bullosa and offers renewed hope for patients and their families.