An exceptionally high number of monogenic disorders lead to incurable blindness, making
them targets for the development of gene-therapy. In order to successfully apply therapeutic …

The first step in vision is the absorption of photons by the photopigments in cone and rod
photoreceptors. After initial amplification within the phototransduction cascade the signal is …

Human retinal organoids from induced pluripotent stem cells (hiPSCs) can be used to
confirm the localization of proteins in retinal cell types and to test transduction and …

Once considered science fiction, gene therapy is rapidly becoming scientific reality, targeting
a growing number of the approximately 250 genes linked to hereditary retinal disorders such …

The present review summarizes the current status of achromatopsia (ACHM) gene therapy‐
related research activities and provides an outlook for their clinical application. ACHM is an …

Background: Gene therapy represents the therapeutic delivery of nucleic acid polymers into
patient cells with the aim of treating an underlying disease. Over the past 2 decades this new …

In vitro retinal organoid modeling from human pluripotent stem cells is becoming more
common place in many ophthalmic laboratories worldwide. These organoids mimic human …

Abstract Treatment approaches for inherited eye diseases require local therapeutic
molecule delivery by intraocular injection. One important factor that can influence the study …

Genome Editing and Transcriptional Activation Using CRISPR-Cas9 Technology, Exploring and
Manipulating Epigenetic Mechanisms in Page 1 GENOME EDITING AND TRANSCRIPTIONAL …

Zusammenfassung Eine Reihe von Netzhauterkrankungen hat bekannte genetische
Ursachen, die prinzipiell durch Gentherapie behandelt werden können. Diese Übersicht …