Development of in vitro gene editing therapy in disease treatment

Abstract

Contemporarily, multitudinous people have been suffering genetic diseases that caused by deficiency of genes. Numerous research has been carried out and many therapies have been proposed for this sort of diseases, such as cell therapy, gene-editing therapy. In vitro gene-editing therapy which refers to both gene-editing and cell therapy has become a promising therapeutic strategy for many genetic diseases. This review mainly focuses on several hereditary diseases relevant to blood, liver and pancreas, systematically summarizes the development of in vitro gene-editing therapy in anemia, congenital liver metabolic diseases and diabetes caused by gene deficiency and discusses the strengthens and limitations of this protocol.