Generation and clinical application of gene modified autologous epidermal sheets in Netherton syndrome - lessons learned from a phase 1 trial.
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DOI: 10.1089/hum.2019.049
Abstract: Netherton Syndrome (NS) is a rare autosomal recessive skin disorder caused by mutations in SPINK5. It is a debilitating condition with notable mortality in the early years of life. There is no curative treatment. We… read more here.
Keywords: gene modified; application gene; netherton syndrome; epidermal sheets ... See more keywords