Use of Replicators in Gene Therapy
Gene therapies offer promising prospects of treating a wide variety of human diseases. In one method, a gene therapy vector can be utilized to deliver an unmutated copy of a gene, called a transgene, to replace a mutated gene in order to treat the genetic disorder. However, lack of expression of a therapeutic transgene and uncontrolled gene silencing are still major obstacles for safety and efficacy of these gene therapy interventions.