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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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Detalles Bibliográficos
Autores principales: Hong, Ying, Casimir, Marina, Houghton, Benjamin C., Zhang, Fang, Jensen, Barbara, Omoyinmi, Ebun, Torrance, Robert, Papadopoulou, Charalampia, Cummins, Michelle, Roderick, Marion, Thrasher, Adrian J., Brogan, Paul A., Eleftheriou, Despina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9073084/
https://www.ncbi.nlm.nih.gov/pubmed/35529868
http://dx.doi.org/10.3389/fimmu.2022.852830