ABSTRACT
The capacity to develop targeted modifications to the human genome has been a major goal in medicine, a goal that has been driven since the discovery of the gene as the basic unit of genetic inheritance. Thus, gene therapy treats a wide range of genetic disorders such as cancer, Duchenne muscular dystrophy, autoimmune diseases, cystic fibrosis, hemophilia, and sickle cell anemia. This therapy became possible with the advancement in technologies such as gene-editing tools like CRISPR-Cas9, CAR-T cell therapy, and bioengineering that facilitates the vectors, which are mostly plasmids or viruses, to deliver genetic material to the specific cell type. However, despite these advancements, several challenges and limitations remain, including complex manufacturing, high costs, immune rejection risks, and ethical concerns surrounding genetic modifications. Furthermore, challenges related to regulatory hurdles, accessibility and cost, and the lack of long-term safety create barriers to the broader application of these treatments. As cell and gene therapies continue to advance, in the future, it is likely that cell and gene therapies will become an integral part of personalized medicine. Researchers can create advanced treatment strategies by combining therapies with other approaches, such as immunotherapy and gene editing. Additionally, the use of advanced technologies such as artificial intelligence and machine learning will play a crucial role in the advancement of cell and gene therapies, enabling the assessment of datasets to identify treatment decisions. This chapter will provide an in-depth examination of the current state of cell and gene therapies, highlighting the key advancements, challenges, and future directions, and exploring the potential of these innovative treatments to transform the landscape of disease treatment.
