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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Methods Clin Dev
Prif Awduron: Carbonaro-Sarracino, Denise A., Chun, Krista, Clark, Danielle N., Kaufman, Michael L., Jin, Xiangyang, Wang, Xiaoyan, Kohn, Donald B.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2021
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7940710/
https://ncbi.nlm.nih.gov/pubmed/33738330
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.007
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