Dystrophic Epidermolysis Bullosa

Arianna Tovar Vetencourt1, Ibrahim Sayed-Ahmed1, Jennifer Gomez1

  • 1From the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami (A.T., I.S.-A., J.G., A.L.S.); and Krystal Biotech, Pittsburgh (H.C., B.A., K.C., T.P., S.K.).

概括

贝雷马基因基因疗法 (B-VEC) 改善了一位患有严重眼部痕的患者的视力. 这种新的治疗方法为这种罕见的遗传疾病中保持视力提供了希望.