Review of Gene Therapy Researches and Clinical Applications

Hanqing Ye

Beyond Biotech, LLC. Shrewsbury, USA & Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, US

Dr. Hanqing Ye, Beyond Biotech, LLC. Shrewsbury, USA & Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, US.

Keywords: Gene Therapy; Clinical Application; Gene Delivery; Genetic Disease; CRISPR-Cas9

Abstract

The application of recombinant DNA technologies in human gene therapy was firstly raised several decades ago, with science advancement, the utilization of genetically modified techniques offered vast therapeutic benefits and potential to these patients with severe genetic diseases, such as muscular dystrophy, B-cell acute lymphoblastic leukemia, cystic fibrosis, etc. Here, the article reviews the history and progress of gene therapy for scientific researches and clinical practices, the strategies of gene therapy design, gene delivery approaches, and state-of-the-art genome editing techniques, CRISPR-Cas9, et al. Although multiple gene therapy products were successively approved by FDA to use in patients, the challenges of gene therapy in the clinical practice are still remaining. The ethical and safety issues of gene therapy is still a consideration in the clinical application, as a consequence, the long-term intervention of germline gene therapy need to be investigated and valuated properly before medical application. The common bottlenecks in gene therapy are still focusing on the level of gene delivery, and both nonviral and viral vector delivery approaches keep limitation in the clinical application and need to be further improved.

This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).