Lentiviral Mediated ADA2 Gene Transfer Corrects the Defects Associated With Deficiency of Adenosine Deaminase Type 2

Lentiviral Mediated ADA2 Gene Transfer Corrects the Defects Associated With Deficiency of Adenosine Deaminase Type 2

Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-allelic loss-of-function mutations in ADA2. Treatment with anti-TNF is effective for the autoinflammatory and vasculitic components of the disease but does not correct marrow failure or immunodeficiency;...

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Bibliographic Details
Main Authors: Ying Hong, Marina Casimir, Benjamin C. Houghton, Fang Zhang, Barbara Jensen, Ebun Omoyinmi, Robert Torrance, Charalampia Papadopoulou, Michelle Cummins, Marion Roderick, Adrian J. Thrasher, Paul A. Brogan, Despina Eleftheriou
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-04-01
Series:Frontiers in Immunology
Subjects:
gene therapy
DADA2
stem cells
macrophages
anti-TNF
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2022.852830/full

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