Abstract
SummaryGene based therapies for inherited retinal dystrophies have improved dramatically over the past 10 years. Clinical trials in patients based on the adeno‐associated virus (AAV) mediated gene transfer of a correct cDNA copy of the mutant gene were started for several disorders, including early onset severe retinal dystrophy associated with mutations in the RPE65 gene, and juvenile retinoschisis due to mutations in the RS1 gene. Furthermore, optogenetic approaches and cell based therapies are on the verge to the clinical application. The highest expectations though have raised the recent development of genome editing strategies based on CRISPR‐Cas endonucleases, which together with a template DNA enable the correction of the mutant DNA sequence ex vivo or in vivo in photoreceptor cells. In this lecture, an overview will be given on the current state of the art in gene based therapies for pediatric patients, and limitations and potential solutions will be discussed.
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