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Author

Pietro De Angeli

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Open access Aug 2026

A mutation-agnostic and allele-specific ASO strategy demonstrates potent functional rescue and retinal preservation in RHO-linked retinitis pigmentosa

Autosomal dominant retinitis pigmentosa (adRP) caused by RHO mutations is a leading form of inherited retinal degeneration. Extensive allelic heterogeneity of RHO pathogenic variants limits the translational applicability of mutation-specific gene therapies. To address this, we developed SNARE (SNP-guided Silencing of...

Salome Spaag, Wen-Hsuan Wu, Justin Yun et al. · 1 citation
#gene editing Review Oct 2026

[Genetic therapies for Stargardt disease].

The diversity of these approaches takes account of the broad spectrum of diseases that ranges from a disease limited to the macula to a generalized rod-cone dystrophy and perspectively opens up individualized treatment strategies depending on the genotype and phenotype.

G. Ansari, Lucas Janeschitz-Kriegl, Pietro De Angeli et al. · 0 citations
Open access Jul 2026

Single-cell analysis reveals impaired Müller glia-mediated intercellular communication and photoreceptor pathology in USH1C retinal organoids

It is demonstrated that disruption of Müller glia signaling contributes to an overall loss of retinal integrity, providing novel insights into USH1C pathogenesis and offering targets for therapeutic interventions.

Nicole Wenck, Mark Zorin, Qiang Wang et al. · 0 citations

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