gene therapy
A therapeutic approach involving introduction of genetic material to correct defective genes causing immunodeficiency diseases.
Full Definition
An emerging treatment modality for primary immunodeficiencies that involves introducing functional genes into a patient's cells to correct underlying genetic defects. Current approaches include ex vivo gene therapy, where the patient's own stem cells are modified with viral vectors containing the normal gene and then reinfused, and in vivo gene editing techniques. Gene therapy has shown particular success in treating severe combined immunodeficiency caused by adenosine deaminase deficiency and X-linked SCID. The field continues to evolve with advances in vector design and gene editing technologies, offering hope for treating previously incurable immunodeficiencies without the need for matched donors.
Usage
Usage note: Distinguish from gene editing; emphasize therapeutic application in immunodeficiency contexts.
In Context
- "Gene therapy protocols offered an alternative to bone marrow transplantation for patients lacking suitable donors." — Clinical trial documentation
- "Long-term follow-up after gene therapy demonstrated sustained immune reconstitution without adverse events." — Research outcome report