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Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders

Abstract Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding retinal dystrophies has become clinical reality. Therapeutically impactful targeting of photoreceptors still relies on subretinal vector delivery, which detaches the retina and harbours substantial risks...

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Autori principali: Marina Pavlou, Christian Schön, Laurence M Occelli, Axel Rossi, Nadja Meumann, Ryan F Boyd, Joshua T Bartoe, Jakob Siedlecki, Maximilian J Gerhardt, Sabrina Babutzka, Jacqueline Bogedein, Johanna E Wagner, Siegfried G Priglinger, Martin Biel, Simon M Petersen‐Jones, Hildegard Büning, Stylianos Michalakis
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Nature 2021-02-01
Serie:EMBO Molecular Medicine
Soggetti:
achromatopsia
intravitreal delivery
novel AAV
retina
Accesso online:https://doi.org/10.15252/emmm.202013392
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