Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

BACKGROUND: Omenn syndrome (OS) is a rare severe combined immunodeficiency associated with autoimmunity and caused by defects in lymphoid-specific V(D)J recombination. Most patients carry hypomorphic mutations in recombination-activating gene (RAG) 1 or 2. Hematopoietic stem cell transplantation is...

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Detaylı Bibliyografya
Yayımlandı:J Allergy Clin Immunol
Asıl Yazarlar: Capo, Valentina, Castiello, Maria Carmina, Fontana, Elena, Penna, Sara, Bosticardo, Marita, Draghici, Elena, Poliani, Luigi P., Sergi Sergi, Lucia, Rigoni, Rosita, Cassani, Barbara, Zanussi, Monica, Carrera, Paola, Uva, Paolo, Dobbs, Kerry, Sacchetti, Nicolò, Notarangelo, Luigi D., van Til, Niek P., Wagemaker, Gerard, Villa, Anna
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2017
Konular:
Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6081264/
https://ncbi.nlm.nih.gov/pubmed/29241731
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jaci.2017.11.015
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