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Advances in Split Vector Approaches for Adeno-Associated Virus Gene Therapy

Feng Gu1,2

  • 1School of Medicine, Hunan Normal University, The Key Laboratory of Model Animals and Stem Cell Biology in Hunan Province, Changsha, China.

Human Gene Therapy
|July 18, 2023
PubMed
Summary

No abstract available in PubMed .

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Gene Therapy00:59

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 inserted. The...