Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine

Here we report, for the first time, the engineering of human red blood cells (RBCs) with an entire metabolic pathway as a potential strategy to treat patients with guanidinoacetate methyltransferase (GAMT) deficiency, capable of reducing the high toxic levels of guanidinoacetate acid (GAA) and resto...

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Detalles Bibliográficos
Autores principales: Bianchi, Marzia, Rossi, Luigia, Pierigè, Francesca, De Angeli, Pietro, Aliano, Mattia Paolo, Carducci, Claudia, Di Carlo, Emanuele, Pascucci, Tiziana, Nardecchia, Francesca, Leuzzi, Vincenzo, Magnani, Mauro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8917272/
https://www.ncbi.nlm.nih.gov/pubmed/35317049
http://dx.doi.org/10.1016/j.omtm.2022.02.007

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