Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a monogenic metabolic disease caused by mutations in the PKLR gene that leads to hemolytic anemia of variable symptomatology and that can be fatal during the neonatal period. PKD recessive inheritance trait and its curative treatment by allogeneic bone marrow tran...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Garcia-Gomez, Maria, Calabria, Andrea, Garcia-Bravo, Maria, Benedicenti, Fabrizio, Kosinski, Penelope, López-Manzaneda, Sergio, Hill, Collin, del Mar Mañu-Pereira, María, Martín, Miguel A, Orman, Israel, Vives-Corrons, Joan-LLuis, Kung, Charles, Schambach, Axel, Jin, Shengfang, Bueren, Juan A, Montini, Eugenio, Navarro, Susana, Segovia, Jose C
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5088764/
https://ncbi.nlm.nih.gov/pubmed/27138040
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.87
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