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Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that le...

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Autori principali: Montiel-Equihua, Claudia A, Thrasher, Adrian J, Gaspar, H Bobby
Natura: Artigo
Lingua:Inglês
Pubblicazione: Dove Medical Press 2009
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3781725/
https://ncbi.nlm.nih.gov/pubmed/24198507
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