Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Pivot Concepts:
Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Target Concepts:
Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Query: UMLS:C0598766 (
leukemogenesis
)
4,065
document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)
The former definition of gene therapy was the infusion of genes or cells transduced with genes into humans for the treatment or prevention of a disease, i.e., gene therapy adds a functional gene into the patients' genome, whereas the mutated gene remains as it is. Because most of the immune immunodeficiency disorders (
PID
) are caused by single gene mutations, this therapeutic option may provide a clinical effect. However, the treatment has a severe problem of
leukemogenesis
caused by insertional mutagenesis; therefore, it is not applicable for diseases caused by the gain-of-function of mutated genes. To address this, gene therapy using gene correction techniques will come to the forefront of the mainstream research. Herein, I have focused on the present outline of gene therapy by gene addition and described the future prospects of gene therapy by gene correction for
PID
.
...
PMID:[Gene therapy for primary immunodeficiency]. 3159 64
