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Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells
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Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy

F Carlucci1, A Tabucchi, A Aiuti

  • 1Dipartimento di Medicina Interna, Scienze Endocrino-Metaboliche e Biochimica, Università di Siena, Siena, Italy.

Nucleosides, Nucleotides & Nucleic Acids
|December 2, 2004
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...