Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders

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 of collat...

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Pubblicato in:EMBO Mol Med
Autori principali: Pavlou, Marina, Schön, Christian, Occelli, Laurence M, Rossi, Axel, Meumann, Nadja, Boyd, Ryan F, Bartoe, Joshua T, Siedlecki, Jakob, Gerhardt, Maximilian J, Babutzka, Sabrina, Bogedein, Jacqueline, Wagner, Johanna E, Priglinger, Siegfried G, Biel, Martin, Petersen‐Jones, Simon M, Büning, Hildegard, Michalakis, Stylianos
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley and Sons Inc. 2021
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Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8033523/
https://ncbi.nlm.nih.gov/pubmed/33616280
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.202013392
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