Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease

Mucopolysaccharidosis type I (MPS-I) is a severe genetic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) enzyme. Ex vivo hematopoietic stem cell (HSC) gene therapy is a promising therapeutic approach for MPS-I, as demonstrated by preclinical studies performed in naive MPS-I mice. Ho...

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Detalles Bibliográficos
Autores principales: Squeri, Giorgia, Passerini, Laura, Ferro, Francesca, Laudisa, Cecilia, Tomasoni, Daniela, Deodato, Federica, Donati, Maria Alice, Gasperini, Serena, Aiuti, Alessandro, Bernardo, Maria Ester, Gentner, Bernhard, Naldini, Luigi, Annoni, Andrea, Biffi, Alessandra, Gregori, Silvia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6612662/
https://www.ncbi.nlm.nih.gov/pubmed/31060789
http://dx.doi.org/10.1016/j.ymthe.2019.04.014

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