The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
本公开涉及增加功能性人 VII 型
胶原蛋白表达的反义寡聚体及相关组合物和方法,以及治疗营养不良性表皮松解症和相关疾病的方法,并涉及诱导排除人 VII 型
胶原蛋白 mRNA 中的外显子 80。