An Introduction To Gene Therapy And Its Applications For Drug Development

Gene therapy involves modifying, manipulating, or deleting genes to treat, prevent, or cure diseases. There are two types of gene therapy: in vivo, which involves directly administering the treatment to the patient, and ex vivo, which involves modifying cells outside the patient's body and then reintroducing them. Gene therapy has shown promise in treating various diseases, particularly cancer and rare diseases. It also has potential applications in treating neuromuscular disorders, blood disorders, ocular disorders, diabetes, osteoarthritis, cardiac arrhythmias, and hearing and balance disorders. However, there are risks and safety concerns associated with gene therapy, including potential adverse side effects and challenges with gene delivery. Developing gene therapies can be complex, costly, and time-consuming, with challenges in creating safe and effective vectors for delivering genes. Organ-on-a-Chip technology offers a solution by providing a platform for better predicting the success and safety of gene therapies. This technology can evaluate transduction efficiency, assess immune responses, and predict hepatotoxicity, leading to improved clinical trials and vector design.