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Gene Therapy

Gene Therapy

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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Updated: Jan 13, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

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Optimizing Gene Therapies for Creatine Transporter Deficiency: Correcting by Connecting

Kyle P Lillis^1,2

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

Epilepsy Currents

|January 9, 2026

Summary

No abstract available in PubMed .

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