International journal of molecular sciencesGuanidinoacetate N-MethyltransferaseAnimalsGenetic TherapyDependovirusMice
A CNS-Directed, AAV9 Gene Therapy Restores Expression and Biochemical Function of Guanidinoacetate Methyltransferase in Models of GAMT Deficiency.
Robyn Binsfeld, Troy Webster, Ilona Tkachyova, Michael Tropak, Melissa Mitchell, Tesla Peretti, Andreas Schulze, Jagdeep S Walia
Published: 202610.3390/ijms27021035
Abstract
Guanidinoacetate methyltransferase (GAMT) is an essential enzyme in the biosynthesis of creatine, an important molecule in energy recycling. GAMT loss of function leads to GAMT deficiency (GAMT-D), an autosomal recessive disorder resulting in low cre…
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