Gene Therapy: Recent Advancement and Challenges
Adamya Aggarwal
Department of Otolaryngology, University of Miami Miller School of Medicine, Miami, FL, USA.
Rahul Mittal *
Department of Otolaryngology, University of Miami Miller School of Medicine, Miami, FL, USA.
*Author to whom correspondence should be addressed.
Abstract
Background: Gene therapy involves delivering therapeutic genomic material to a target tissue to modify expression of a protein or induce other characteristic changes. Recent advancements in the field, including FDA-approval of numerous gene therapies, are paving the way for future technological progress. While gene therapy can be either somatic or germline, most research and drug development has focused on somatic cells. In this article, we discuss the recent advancements and challenges associated with gene therapy.
Main Text: There are multiple types of gene therapies, including hematopoietic stem cell therapy, CAR-T cell therapy, and Crispr/Cas9 gene therapy. Rare diseases and cancers are being researched to determine methods of treatment using gene therapy, and several clinical trials have been performed within the last decade to test the efficacy of new therapeutic drugs, many of them at least somewhat successful. However, gene therapy does pose some challenges, including large-scale manufacturing of vectors, precision of gene delivery to target tissue, and, most importantly, the immune responses of patients.
Conclusions: The near future is an exciting time for new gene therapy technologies and strategies. Researchers and patients can look forward to new advancements in base editing, prime editing, and RNA-targeted editing technologies. Furthermore, future research can focus on new genetic targets, such as genes whose functions may still be unknown and epigenomic elements.
Keywords: Gene therapy, stem cell therapy, rare diseases, gene editing