Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are impaired. Gene therapy (GT) with an autologous CD34(+)-enriched cell fraction that contains CD34(+) cells transduced with a retroviral vector...

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Vydáno v:Mol Ther
Hlavní autoři: Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, Aiuti, Alessandro
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910668/
https://ncbi.nlm.nih.gov/pubmed/29433935
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.022
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