Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9

Citrullinemia type I is a rare autosomal-recessive disorder caused by deficiency of argininosuccinate synthetase (ASS1). The clinical presentation includes the acute neonatal form, characterized by ammonia and citrulline accumulation in blood, which may lead to encephalopathy, coma, and death, and t...

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Detalles Bibliográficos
Autores principales: Lisjak, Michela, Iaconcig, Alessandra, Guarnaccia, Corrado, Vicidomini, Antonio, Moretti, Laura, Collaud, Fanny, Ronzitti, Giuseppe, Zentilin, Lorena, Muro, Andrés F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10514469/
https://www.ncbi.nlm.nih.gov/pubmed/37744006
http://dx.doi.org/10.1016/j.omtm.2023.08.022

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10514469/
https://www.ncbi.nlm.nih.gov/pubmed/37744006
http://dx.doi.org/10.1016/j.omtm.2023.08.022

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