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Phenotype reversion as “natural gene therapy” in Fanconi anemia by a gene conversion event

Somatic mosaicism appears as a recurrent phenomenon among patients suffering from Fanconi anemia (FA), but its direct prognostic significance mostly remains an open question. The clinical picture of FA mosaic subjects could indeed vary from just mild features to severe hematologic failure. Here, we...

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Detalles Bibliográficos
Autores principales: Persico, Ilaria, Fiscarelli, Ilaria, Pelle, Alessandra, Faleschini, Michela, Pasini, Barbara, Savoia, Anna, Bottega, Roberta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10544911/
https://www.ncbi.nlm.nih.gov/pubmed/37790699
http://dx.doi.org/10.3389/fgene.2023.1240758