The Current Status of Gene Therapy for the Treatment of Cancer

Abstract

Gene therapy is the administration of foreign genomic material into the host tissue to modify the expression of a gene product or to change the biological properties of cells for therapeutic use. Initially, the major objective of gene therapy was to manage genetic diseases, but now different disorders with several patterns of acquired and inherited disorders are targets of gene therapy. Over three decades, the advancement of Genome engineering technologies facilitated gene therapy for the prevention and management of intractable diseases. Researchers are advancing with cautious optimism that safe and effective treatment will give to patients with single-gene disorders and complex acquired disorders. To date, over 3000 genes associates with disease-causing mutations, and about 2600 gene therapy trials are undergoing for the management of various disorders. This review summarizes the principles of genome-editing approaches, such as zinc finger nucleases, transcription activator-like effector nucleases, meganucleases, and the CRISPR/Cas9 system with the underlying mechanisms. This review also explains the types of gene delivery systems as viral [adenoviral, adeno association, herpes simplex virus] and nonviral delivery systems (physical: DNA bombardment, electroporation) and (chemical: Cationic lipids, cationic polymers). Finally, this review summarizes gene therapy medicines approved to treat cancer in detail, including names, indications, vectors, and mode of gene therapy. Gene therapy becomes an alternative to an existing management for different diseases. Therefore, gene products with safe vectors and better biotechnologies play a significant role in the prophylaxis and management of various disorders in the future.

Gene Therapy Targeting p53 and KRAS for Colorectal Cancer Treatment: A Myth or the Way Forward?

“…There has been good progress in gene therapy for cancer in the last three decades, wherein a number of drugs have been approved and many are still at the stage of clinical trials. In the future, tumor genomic profiling coupled with the analysis of host humoral and cellular immunity will ensure the selection of the most appropriate patient and treatment modalities for gene therapy [ 62 ].…”

LncRNA MORT (ZNF667-AS1) in Cancer—Is There a Possible Role in Gynecological Malignancies?

“…Genetic and epigenetic factors have been shown to have a central role in the development of cancer. Gene therapy approaches have been developed, and these include suicide gene therapy, anti-angiogenic gene therapy, siRNA therapy, immunotherapy, oncolytic virotherapy, pro-apoptotic gene therapy, and gene-directed-enzyme prodrug therapy [93,94].…”

Dendrimers as Non-Viral Vectors in Gene-Directed Enzyme Prodrug Therapy

“…Transport vectors contribute, sometimes decisively, to the effectiveness of this therapeutic strategy [ 48 ]. Numerous strategies and tools have been used for the intracellular delivery of nucleic acids, including viral (VV) and non-viral vectors (nVV), with the latter being represented by chemical vectors (cationic lipids, lipid polymers, cationic polymers) and physical vectors obtained by different methods (DNA bombardment, ultrasound, electroporation, sonoporation, magnetofection) [ 49 , 50 , 51 ].…”

“…The use of revolutionary techniques focusing on clustered regularly interspaced short palindromic repeats (CRISPR) is becoming more and more evident. The CRISPR/Cas9 system has already been customized to provide new defense capabilities to the host organism, either by modifying the host genome or by directly targeting the factors used [ 50 , 52 ]. The CRISPR/Cas9 system offers great therapeutic potential.…”