The use of gene-modified cells as a source of hematopoietic stem cell transplantation (HSCT) has grown significantly in the last 10 years. Although advances in supportive care, prevention of infections, improved HLA typing, and improved conditioning regimens have improved survival, HSCT can be complicated in those who lack well-matched donors or who have preexisting infections.
The ability to develop viral vectors that stably integrate modified genes into cells occurred in the 1980s and 1990s. The first gene therapy trial occurred with a 4-year-old girl with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). The patient received several infusions of a gamma retroviral vector containing normal ADA cDNA which led to endogenous production of ADA.
The sequence of events for gene modification therapy is as follows:
- 1.
Stem cells collected from affected patient.
- 2.
Stem cells cultured in vitro with modified virus carrying the corrective gene sequence.
- 3.
Precondition...
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Leiding, J.W. (2019). Management of Immunodeficiency: Gene Therapy. In: MacKay, I., Rose, N. (eds) Encyclopedia of Medical Immunology. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-9209-2_83-1
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