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

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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Bibliographic Details
Main Authors: Ilaria Persico, Ilaria Fiscarelli, Alessandra Pelle, Michela Faleschini, Barbara Pasini, Anna Savoia, Roberta Bottega
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-09-01
Series:Frontiers in Genetics
Subjects:
Fanconi anemia
gene conversion
mosaicism
natural gene therapy
phenotype reversion
Online Access:https://www.frontiersin.org/articles/10.3389/fgene.2023.1240758/full

Internet

https://www.frontiersin.org/articles/10.3389/fgene.2023.1240758/full

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