Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders.

ABSTRACT: The field of gene therapy is striving more than ever to define a path to the clinic and the market. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide. These advances raise great hope to treat devastating rare and inherited diseases as well as incurable illnesses. Understanding of the precise pathomechanisms of diseases as well as the development of efficient and specific gene targeting and delivery tools are revolutionizing the global market. Currently, human cancers and monogenic disorders are indications number one. The elevated prevalence of genetic disorders and cancers, clear gene manipulation guidelines and increasing financial support for gene therapy in clinical trials are major trends. Gene

SUBMITTER: Shahryari A 

PROVIDER: S-EPMC6773888 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature