Alt text: Graphic with the headline “Did You Know?” stating that over 300 million people live with rare diseases and only 0.025%–0.05% have choroideremia (CHM), followed by the message “Even the rarest voices deserve to be heard.” Includes the Rare Disease Day logo, the date February 28, and a globe outline.

Today is Rare Disease Day! 🌍 Over 300M people live with a rare disease. CHM affects just 1 in 50K–100K. Support the ultra-rare CHM community & fund sight-saving research: secure.qgiv.com/for/choroide...

#RareDiseaseDay #CureCHM #SightSavingResearch

Klinische Endpunkte und translationale Herausforderungen - Die Ophthalmologie Background Optogenetic vision-restorative approaches have entered the clinical stage of development. Objective This article provides an overview of the current status of translation. It discusses spec...

Our new review article: clinical endpoints & translational challenges in #optogenetics for vision restoration. From patient expectations to #ClinicalTrial endpoints. First review to focus specifically on clinical perspective. doi.org/10.1007/s003...

#retina #GeneTherapy
@unimarburg.bsky.social

Advanced #CHM or RP can permanently destroy photoreceptors. Researchers are developing vision-restoration strategies like #Optogenetics. In an interview study patients are invited to talk about their expectations, needs & rehab/assistive gaps. lindnerlab.de/index.php/ne... #RareDisease #GeneTherapy

We have created an (exhaustive - we think) directory of interventional clinical trials on optogenetic visual restoration: lindnerlab.de/index.php/pu.... Hopefully a helpful resource for funders, researchers and regulators!

#retina #GeneTherapy #optogenetics
@unimarburg.bsky.social

Delighted to see some more of our latest research online at BioRXiv: www.biorxiv.org/content/10.6...
We generated and characterized a mouse model for KCNV2 related retinal dystrophy. It nicely resembles key characteristics of the human disease phenotype.
#retina #GeneTherapy @unimarburg.bsky.social

Lipid nanoparticles for retinal gene delivery - Read out latest preprint at biorxiv.org/content/10.1101/2025.11.21.689792v1 !
LNPs are able to transfect a variety of retinal cell types - including NEURONS! Previous ILM peeling improves efficacy!
#retina #GeneTherapy #LNP @unimarburg.bsky.social

The ERGtools2 package: a toolset for processing and analysing visual electrophysiology data - Documenta Ophthalmologica Purpose To introduce ERGtools2, an open-source R package for processing, analysing and long-term storing visual electrophysiology data. Methods A dataset comprising Electroretinogram (ERG) recordings ...

ERGtools2 — an #OpenSource R package for analyzing and managing visual electrophysiology data ( #ERG / #VEP ).
Device-independent, reproducible, FAIR-compliant & ready for preclinical and clinical research.
Paper: doi.org/10.1007/s106...
Code: github.com/moritzlindne...
@unimarburg.bsky.social

New perspectives on #optogenetic #VisionRestoration
In my letter in @moltherapy.bsky.social‬, I comment on recent clinical data on vMCO-010 and highlight the need for mechanistic insight & objective efficacy measures.
Many open questions remain - exciting times ahead! @unimarburg.bsky.social #Retina

Optogenetic Therapy in patients with residual visual function?
In this study, we show that optogenetic responses complexly interact with native visual processing. doi.org/10.1172/jci.... @unimarburg.bsky.social @ox.ac.uk @jci-insight.bsky.social ‬ #Optogenetics #Neuroscience #Retina #GeneTherapy