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Nature Reviews. Drug Discovery Mar 2021
Topics: Animals; Dependovirus; Genetic Therapy; Genetic Vectors; Humans
PubMed: 33495615
DOI: 10.1038/d41573-021-00017-7 -
European Journal of Internal Medicine Jan 2014Gene therapy, replacing a defective gene by a functional copy, has been in development for more than 40years. Initial efforts involved engineering viral vectors to... (Review)
Review
BACKGROUND
Gene therapy, replacing a defective gene by a functional copy, has been in development for more than 40years. Initial efforts involved engineering viral vectors to deliver genes to the appropriate cells. Early successes in severe combined immunodeficiency (SCID) were later derailed by safety issues including host reaction to the vector and gene insertion near promoters that favored secondary leukemia.
METHODS
Systematic review of the literature using PubMed.gov with key word gene therapy from 1972 to March 2013. Google search with key word gene therapy.
RESULTS
Despite early setbacks, progresses for monogenic diseases continued unabated. Patients with SCIDs have been cured and the first gene therapy has been approved for lipoprotein lipase deficiency. Many clinical research studies are ongoing as part of systematic clinical development program with a view to have more gene therapies approved.
CONCLUSION
Our review highlights progresses and questions that remain to be answered to make gene therapy an integral part of our therapeutic arsenal.
Topics: Adrenoleukodystrophy; Dependovirus; Genetic Therapy; Genetic Vectors; Hemophilia B; Humans; Hyperlipoproteinemia Type I; Lentivirus; Retroviridae; Severe Combined Immunodeficiency
PubMed: 24129166
DOI: 10.1016/j.ejim.2013.09.009