Abstract：
UNASSIGNED: Dystrophic epidermolysis bullosa (DEB), a rare genetic skin disease caused by loss-of-function mutations in COL7A1, the gene encoding type VII collagen (COL7), is characterized by skin blistering, scarring, and extracutaneous manifestations that markedly reduce patient quality-of-life. Beremagene geperpavec-svdt (\'B-VEC\') is a gene therapy employing a non-integrating, replication-defective herpes simplex virus type 1 (HSV-1)-based vector encoding two copies of full-length human COL7A1 to restore COL7 protein after topical administration to DEB wounds. B-VEC was approved in the United States in 2023 as the first topical gene therapy and the first approved treatment for DEB. However, few providers have experience with use of this gene therapy.
UNASSIGNED: Data was obtained through literature review and the experience of providers who participated in the B-VEC clinical study or initiated treatment after B-VEC approval.
UNASSIGNED: This review discusses the burden of disease, describes the clinical trial outcomes of B-VEC, and provides physician and patient/caregiver recommendations as a practical guide for the real-world use of B-VEC, which can be administered in-office or at the patient\'s home.
UNASSIGNED: By continuing to optimize the practical aspects of B-VEC administration, the focus will continue to shift to patient-centric considerations and improved patient outcomes.
UNASSIGNED: Data was obtained through literature review and the experience of providers who participated in the B-VEC clinical study or initiated treatment after B-VEC approval.
UNASSIGNED: This review discusses the burden of disease, describes the clinical trial outcomes of B-VEC, and provides physician and patient/caregiver recommendations as a practical guide for the real-world use of B-VEC, which can be administered in-office or at the patient\'s home.
UNASSIGNED: By continuing to optimize the practical aspects of B-VEC administration, the focus will continue to shift to patient-centric considerations and improved patient outcomes.
摘要：
■营养不良性大疱性表皮松解症（DEB），一种罕见的遗传性皮肤病，由编码VII型胶原（COL7）的基因COL7A1功能丧失突变引起，以皮肤起泡为特征，疤痕,和显著降低患者生活质量的皮外表现。Beremagenegeperpavec-svdt（\'B-VEC\'）是一种采用非整合性，基于复制缺陷型单纯疱疹病毒1型（HSV-1）的载体，编码两个全长人COL7A1的拷贝，以在局部施用至DEB伤口后恢复COL7蛋白。B-VEC于2023年在美国被批准为第一个局部基因治疗和DEB的第一个批准治疗。然而,很少有提供者有使用这种基因疗法的经验。
■数据是通过文献综述和参与B-VEC临床研究或在B-VEC批准后开始治疗的提供者的经验获得的。
■这篇综述讨论了疾病负担，描述了B-VEC的临床试验结果，并提供医生和患者/护理人员的建议，作为实际使用B-VEC的实用指南，可以在办公室或在患者家中进行管理。
■通过继续优化B-VEC管理的实际方面，重点将继续转移到以患者为中心的考虑和改善患者预后.
■数据是通过文献综述和参与B-VEC临床研究或在B-VEC批准后开始治疗的提供者的经验获得的。
■这篇综述讨论了疾病负担，描述了B-VEC的临床试验结果，并提供医生和患者/护理人员的建议，作为实际使用B-VEC的实用指南，可以在办公室或在患者家中进行管理。
■通过继续优化B-VEC管理的实际方面，重点将继续转移到以患者为中心的考虑和改善患者预后.
