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Allometric-like scaling of AAV gene therapy for systemic protein delivery

The use of adeno-associated virus (AAV) as a gene delivery vehicle for secreted peptide therapeutics can enable a new approach to durably manage chronic protein insufficiencies in patients. Yet, dosing of AAVs have been largely empirical to date. In this report, we explore the dose-response relation...

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Detalles Bibliográficos
Autores principales: Burr, Alexandra, Erickson, Patrick, Bento, Raphaela, Shama, Kariman, Roth, Charles, Parekkadan, Biju
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9641010/
https://www.ncbi.nlm.nih.gov/pubmed/36381306
http://dx.doi.org/10.1016/j.omtm.2022.10.011