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Formulation of synthetic vectors for cystic fibrosis gene therapy

John Marshall1, Seng H Cheng

  • 1Genzyme Corporation, Framingham, MA, USA.

Methods in Molecular Medicine
|March 29, 2002
PubMed
Abstract

No abstract available in PubMed .

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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...