Advanced Gene Therapy in the Clinical Management

The human genome consists of a genetic code responsible for the transcription of the protein needed by cells for its living. These genes never get altered; however, it happens in certain cases which lead to serious disease conditions. Hence, gene therapy also called “living drug therapy” is administered to correct these gene altercations. After conducting 1700 clinical trials, the promise made by scientists decades ago about delivering a complete cure via gene therapy is worth exploring. Genes are mainly delivered to the subject through vectors, which include both viral and nonviral. A few problems associated with viral vector delivery such as immune reaction, off-target effect, and ineffective delivery have raised skepticism in the gene therapy process. However, with the joint effort of pharmaceutical industries and national institutes, controlling authorities have been established to monitor efficient vector preparation. In addition, with the discovery of nearly 12 gene therapy drugs for various conditions 386 such as cancer, neurologic genetic disorders, and hereditary immune disorder have once again brought gene therapy into the public eye. This chapter here discusses the strategies, modes for gene delivery, CRISPR/ CAS9 technology and its use in gene therapy, clinical application, and the challenges of gene therapy.