Gene therapy for rare genetic disorders

Abstract

Genetic disorders are widespread. However, many of these genetic illnesses have minimal information, making therapy difficult. Gene therapy has emerged as a major breakthrough in the field of medical science to treat diseases resulting from defective genes. The FDA has received over 900 gene therapies for hereditary disorders. Rare genetic illnesses are life threatening and impact 5 per 10000 people worldwide. Researchers are conducting clinical trials to investigate treatments for rare genetic disorders despite a paucity of knowledge. With sufficient clinical trial data showing signs of improvement among the patients and safety of gene delivery systems, gene therapy can be the most efficient treatment strategy for rare genetic disorders. In this review paper, a number of rare genetic disorders and the prospects of gene therapy in the treatment of those disorders were discussed.