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

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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Bibliographic Details
Main Authors: Denise A. Carbonaro-Sarracino, Krista Chun, Danielle N. Clark, Michael L. Kaufman, Xiangyang Jin, Xiaoyan Wang, Donald B. Kohn
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV vector
gene therapy
lentiviral vector
ADA-SCID
ADA-deficiency
inborn errors of metabolism
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012100022X

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