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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...
Autores principales: | Burr, Alexandra, Erickson, Patrick, Bento, Raphaela, Shama, Kariman, Roth, Charles, Parekkadan, Biju |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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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 |
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