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Inklusion ist als Thema aus dem deutschen Bildungssystem nicht mehr wegzudenken und trotzdem stellt sie weiterhin eine Herausforderung auf unterschiedlichen Ebenen dar. Die Qualifizierung der pädagogischen Fachkräfte ist dabei neben der Bereitstellung adäquater Rahmenbedingungen als ein besonders wichtiges Handlungsfeld zu betrachten. Die Bände der Reihe "Qualifizierung für Inklusion" greifen den bestehenden Forschungs- und Entwicklungsbedarf auf und geben einen Überblick über die Ergebnisse der vom BMBF im Rahmen des Programms "Qualifizierung der pädagogischen Fachkräfte für inklusive Bildung" geförderten Forschungsprojekte. Adressiert werden damit sowohl Wissenschaftler:innen als auch mit dem Themenfeld Inklusion befasste Personen und Institutionen der Aus-, Fort- und Weiterbildung, der Bildungsadministration und der Bildungspolitik. Der vierte Band der Reihe stellt die Ergebnisse und Materialien der Projekte vor, die zur Qualifizierung für Inklusion in der Berufsschule, Hochschule und der Erwachsenenbildung gearbeitet haben. Die Reihe besteht aus drei weiteren Bänden, in denen die Ergebnisse zur Qualifizierung für Inklusion im Elementarbereich (Band 1), in der Grundschule (Band 2) sowie in der Sekundarstufe (Band 3) vorgestellt werden.

Blindness. --- Amaurosis --- Vision disorders

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Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequences of these defects are rapidly emerging, providing hope for personalized medicine. ‘Classical’ gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM, for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach, but also opened avenues for the development of other strategies. However, every gene—or even every mutation—may need a tailor-made therapeutic approach, in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy, other subtypes of molecular therapy are currently being designed and/or implemented, including splice modulation, DNA or RNA editing, optogenetics and pharmacological modulation. In addition, the development of proper delivery vectors has gained strong attention, and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue, we aim to describe the current state of the art of molecular therapeutics for IRD, and discuss existing and novel therapeutic strategies, from idea to implementation, and from bench to bedside.

induced pluripotent stem cell (iPSC) --- clustered regularly interspaced short palindromic repeats (CRISPR) --- homology-directed repair (HDR) --- Enhanced S-Cone Syndrome (ESCS) --- NR2E3 --- AAV --- retina --- gene therapy --- dual AAV --- gold nanoparticles --- DNA-wrapped gold nanoparticles --- ARPE-19 cells --- retinal pigment epithelium --- clathrin-coated vesicles --- endosomal trafficking --- retinitis pigmentosa --- autosomal dominant --- G56R --- putative dominant negative effect --- gapmer antisense oligonucleotides --- allele-specific knockdown --- Leber congenital amaurosis and allied retinal ciliopathies --- CEP290 --- Flanders founder c.4723A &gt --- T nonsense mutation --- Cilia elongation --- spontaneous nonsense correction --- AON-mediated exon skipping --- microRNA --- photoreceptors --- rods --- cones --- bipolar cells --- Müller glia --- retinal inherited disorders --- retinal degeneration --- antisense oligonucleotides --- Stargardt disease --- inherited retinal diseases --- splicing modulation --- RNA therapy --- ABCA4 --- iPSC-derived photoreceptor precursor cells --- cyclic GMP --- apoptosis --- necrosis --- drug delivery systems --- translational medicine --- Usher syndrome --- Leber congenital amaurosis --- RPE65 --- nonprofit --- patient registry --- translational --- protein trafficking --- protein folding --- protein degradation --- chaperones --- chaperonins --- heat shock response --- unfolded protein response --- autophagy --- therapy --- IRD --- DNA therapies --- RNA therapies --- compound therapies --- clinical trials --- Retinitis Pigmentosa GTPase Regulator --- adeno-associated viral --- Retinitis Pigmentosa (RP) --- choroideremia --- REP1 --- inherited retinal disease --- treatment --- apical polarity --- crumbs complex --- fetal retina --- PAR complex --- retinal organoids --- retinogenesis --- gene augmentation --- adeno-associated virus (AAV) --- n/a --- Müller glia