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

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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Bibliographic Details
Main Authors: Marzia Bianchi, Luigia Rossi, Francesca Pierigè, Pietro De Angeli, Mattia Paolo Aliano, Claudia Carducci, Emanuele Di Carlo, Tiziana Pascucci, Francesca Nardecchia, Vincenzo Leuzzi, Mauro Magnani
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
metabolic engineering
guanidinoacetate methyltransferase (GAMT)
GAMT deficiency
methionine adenosyl transferase
creatine deficit
RBC loading
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000237

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