New frontiers to cure Alport syndrome: COL4A3 and COL4A5 gene editing in podocyte-lineage cells

Alport syndrome (AS) is an inherited genetic disorder characterized by range of alterations from glomerular basement membrane abnormalities up to end-stage renal disease. Pathogenic variants in the collagen α3, α4, and α5 encoding genes are causative both of the autosomal dominant and of the X-linke...

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Vydáno v:Eur J Hum Genet
Hlavní autoři: Daga, Sergio, Donati, Francesco, Capitani, Katia, Croci, Susanna, Tita, Rossella, Giliberti, Annarita, Valentino, Floriana, Benetti, Elisa, Fallerini, Chiara, Niccheri, Francesca, Baldassarri, Margherita, Mencarelli, Maria Antonietta, Frullanti, Elisa, Furini, Simone, Conticello, Silvestro Giovanni, Renieri, Alessandra, Pinto, Anna Maria
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer International Publishing 2019
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7080842/
https://ncbi.nlm.nih.gov/pubmed/31754267
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41431-019-0537-8
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