"KaDee Troop is the mother of seven adopted children, four of whom have a rare genetic disorder that causes their skin to blister and tear at the slightest touch. Wounds heal slowly if at all and with each re-injury, the skin becomes thinner and more fragile. For most of the children's lives, hope came in the form of gauze and bandages."
"With the gene-therapy safety net, one of the children, Jayne, now 14, feels confident enough to perform backflips on the trampoline; Alex, at 12, barrels down rocky trails on his mountain bike; Ellen, 13, has gained independence, doing wound care by herself; and Zack, 22, lifts weights using a custom arm prosthetic with dumbbell-clamping grips and a shoulder harness fitted over his skin that was once unable to bear the strain."
"That is, until 2023, when it arrived in a bottle. That year, US regulators approved the first gene therapy for a form of epidermolysis bullosa, the condition that affects the Troop children and is sometimes referred to as butterfly skin. All four children began applying the gel-based treatment to their raw, vulnerable lesions; the results were profound. Wounds that had stubbornly resisted healing for years began to close."
Four children with epidermolysis bullosa experienced chronic, fragile skin that blistered and tore from minimal contact, with wounds that healed slowly or not at all. In 2023, regulators approved a gel-based gene-replacement therapy, beremagene geperpavec (B-VEC), for a form of the disease. The topical treatment applied at home produced profound healing of long-standing lesions and enabled children to perform physical activities, gain independence in wound care and use prosthetics. The therapy represents the first approved gene-replacement treatment for a non-cancerous skin disorder and the first topical gene therapy for at-home use.
Read at www.nature.com
Unable to calculate read time
Collection
[
|
...
]