Gene Therapy for LCA 6

This chapter discusses the genetic disorder Lieber congenital amorosis (LCA) and specifically focuses on LCA 6. It explores the RP group one gene mutations associated with LCA and the potential use of gene therapy, specifically AV capsid ANK-80, to deliver a functional copy of the gene into retinal cells. The chapter also touches on the current stage of the gene therapy product in the pipeline and discusses the potential long-term effects of gene therapies.