National Human Genome Research Institute (NHGRI)

Gene therapy is an experimental technique for treating disease by altering the patient’s genetic material. Most often, gene therapy works by introducing a healthy copy of a defective gene into the patient’s cells.

Gene therapy is an experimental form of treatment that uses gene transfer of genetic material into the cell of a patient to cure the disease. The idea is to modify the genetic information of the cell of the patient that is responsible for a disease, and then return that cell to normal conditions. Transfer of genetic material is done commonly by using viral vectors that use their own biological capacities to enter the cell and deposit the genetic material. Both inherited genetic diseases and acquired disorders can be treated with gene therapy. Examples of these disorders are primary immune deficiencies, where gene therapy has been able to fully correct the presentation of patients, and/or cancer, where the gene therapy is still at the experimental stage.

Fabio Candotti, M.D.

OccupationSenior Investigator, Genetics and Molecular Biology Branch; Head, Disorders of Immunity Section

BiographyDr. Candotti’s laboratory studies the molecular basis of inherited disorders of the immune system in order to develop better treatments for these conditions. Currently, for many inherited immune deficiency disorders, the only available therapeutic option is hematopoietic stem cell transplantation (HSCT), an intensive procedure that carries a number of risks. Dr. Candotti is seeking treatment alternatives to HSCT, focusing on gene replacement approaches. His laboratory is developing gene therapies for two rare immune deficiency syndromes, adenosine deaminase (ADA) deficiency and Wiskott-Aldrich syndrome (WAS).

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National Human Genome Research Institute (NHGRI)

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