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Optimizing Gene Therapies for Creatine Transporter Deficiency: Correcting by Connecting

Kyle P Lillis1,2

  • 1Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.

Epilepsy Currents
|January 9, 2026
PubMed
Summary

No abstract available in PubMed .

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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