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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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Related Experiment Video

Updated: Mar 8, 2026

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

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

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Delivering efficient liver-directed AAV-mediated gene therapy

J Baruteau^1,2,3, S N Waddington^3,4, I E Alexander^5,6

¹Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, UK.

|January 13, 2017

Summary

No abstract available in PubMed .

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