When a patient receives gene therapy, genetic material is inserted into their own cells to replace any lost or improper functions brought on by cancer or genetic mutations, or to restore those functions. Only a small number of the 7,000 or so rare diseases that have been found have licenced medicines, according to the National Center for Advancing Translational Sciences. More than 80% of rare diseases have a known monogenic (single-gene) aetiology, which has led to tremendous progress in the development of gene therapy for these conditions. Small molecule medications frequently just address the surface-level causes of the disease rather than treating them. To control the illness, this necessitates frequent medicine administration. Gene Therapy for Rare Disease has the capacity to fix underlying genetic flaws.
