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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Publicado no:J Allergy Clin Immunol
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
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Article
Acesso em linha: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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