Mouse study shows gene therapy may correct creatine deficiency disorder

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Mouse study shows gene therapy may correct creatine deficiency disorder

A new study in mice finds that a gene therapy developed by a UCLA researcher appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures. The treatment potentially could represent an improvement upon available therapy for the inheritable disorder, known as guanidinoacetate methyltransferase deficiency (GAMT).

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