Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we engineered myotropic adeno-associated viral (AAV) vectors via a semirational, combinatorial approach that merges AAV capsid...

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Detalles Bibliográficos
Autores principales: El Andari, Jihad, Renaud-Gabardos, Edith, Tulalamba, Warut, Weinmann, Jonas, Mangin, Louise, Pham, Quang Hong, Hille, Susanne, Bennett, Antonette, Attebi, Esther, Bourges, Emanuele, Leborgne, Christian, Guerchet, Nicolas, Fakhiri, Julia, Krämer, Chiara, Wiedtke, Ellen, McKenna, Robert, Guianvarc’h, Laurence, Toueille, Magali, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico, VandenDriessche, Thierry, Agbandje-McKenna, Mavis, Müller, Oliver J., Chuah, Marinee K., Buj-Bello, Ana, Grimm, Dirk
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2022
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9491714/
https://www.ncbi.nlm.nih.gov/pubmed/36129972
http://dx.doi.org/10.1126/sciadv.abn4704

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