Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy

The majority of inherited retinal diseases (IRDs) are caused by mutations in genes expressed in photoreceptors (PRs). The ideal vector to address these conditions is one that transduces PRs in large areas of retina with the smallest volume/lowest titer possible, and efficiently transduces foveal con...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Boye, Sanford L., Choudhury, Shreyasi, Crosson, Sean, Di Pasquale, Giovanni, Afione, Sandra, Mellen, Russell, Makal, Victoria, Calabro, Kaitlyn R., Fajardo, Diego, Peterson, James, Zhang, Hangning, Leahy, Matthew T., Jennings, Colin K., Chiorini, John A., Boyd, Ryan F., Boye, Shannon E.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7264435/
https://ncbi.nlm.nih.gov/pubmed/32304666
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.04.002
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