Abstract
In the study reported, we prove that mutations in the SLC6A8 gene are responsible for SLC6A8 deficiency, a cerebral creatine deficiency syndrome (CCDS), since overexpression of the wild-type SLC6A8 open reading frame (ORF) restores the creatine uptake profile in SLC6A8-deficient fibroblasts.
Publication types
-
Research Support, Non-U.S. Gov't
MeSH terms
-
Amino Acid Transport Disorders, Inborn / genetics
-
Amino Acid Transport Disorders, Inborn / metabolism*
-
Brain Diseases, Metabolic, Inborn / genetics
-
Brain Diseases, Metabolic, Inborn / metabolism*
-
Cells, Cultured
-
Creatine / metabolism*
-
Fibroblasts / drug effects
-
Fibroblasts / metabolism*
-
Guanidines / pharmacology
-
Humans
-
Mutation
-
Nerve Tissue Proteins / antagonists & inhibitors
-
Nerve Tissue Proteins / deficiency
-
Nerve Tissue Proteins / genetics
-
Nerve Tissue Proteins / metabolism*
-
Open Reading Frames / genetics
-
Plasma Membrane Neurotransmitter Transport Proteins / antagonists & inhibitors
-
Plasma Membrane Neurotransmitter Transport Proteins / deficiency
-
Plasma Membrane Neurotransmitter Transport Proteins / genetics
-
Plasma Membrane Neurotransmitter Transport Proteins / metabolism*
-
Propionates / pharmacology
-
Transfection
Substances
-
Guanidines
-
Nerve Tissue Proteins
-
Plasma Membrane Neurotransmitter Transport Proteins
-
Propionates
-
SLC6A8 protein, human
-
Creatine
-
guanidinopropionic acid