Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

Adenosine deaminase (ADA) deficiency is an inborn error of metabolism affecting multiple systems and causing severe combined immunodeficiency. We tested intravenous administration of recombinant adeno-associated virus (AAV) 2/8-ADA vector in ADA-deficient neonate and adult mice or as part of a bimod...

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Detalles Bibliográficos
Autores principales: Carbonaro-Sarracino, Denise A., Chun, Krista, Clark, Danielle N., Kaufman, Michael L., Jin, Xiangyang, Wang, Xiaoyan, Kohn, Donald B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7940710/
https://www.ncbi.nlm.nih.gov/pubmed/33738330
http://dx.doi.org/10.1016/j.omtm.2021.02.007

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