Gene therapy entails inserting genetic material into patients' cells to provide new functions or restore missing or ineffective functions caused by cancer or gene mutations. Although approximately 7,000 rare diseases have been identified, only a few hundred have approved treatments, according to the National Center for Advancing Translational Sciences. Furthermore, because more than 80% of rare diseases have a known monogenic (single-gene) cause, the development of gene therapy for rare diseases is gaining traction. Small molecule drugs frequently treat only symptoms rather than the underlying cause of the disease. Gene Therapy for Rare Disease Market manage the condition, the drugs must be administered on a regular basis. Gene therapy has the potential to repair underlying genetic flaws.
