Inherited blindness is the leading cause of vision loss in the working populations. Recently, the first gene therapy for the treatment of retinal dystrophy caused by mutations in the RPE65 gene became available. This gene therapy is based on adeno-associated virus (AAV) vectors injected under the retina. Subretinal injection is…
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A team of researchers has shown that injection of cholangiocyte organoids in human livers ex vivo can repair the organs’ bile ducts. In this article, Dr Fotios Sampaziotis explains how his team’s study provides the first proof-of-principle for the efficacy of cellular therapies using organoids in human.