somatic gene therapy

Somatic gene therapy involves introducing a normal gene into a person's body cells to correct a mutation causing a genetic disease. This approach aims to repair the defective gene within the individual's genome, potentially restoring normal function.12

While somatic gene therapy can alleviate disease symptoms in the treated person, these genetic modifications are not passed on to future generations. This is in contrast to germline gene therapy, which targets reproductive cells and would allow for heritable genetic changes, though it has only been achieved experimentally in animals.12

Current research focuses on effective delivery systems, often using modified viruses called viral vectors, and ensuring the safety and efficacy of the procedure. Ethical considerations and safety concerns, including potential immune responses and the risk of new mutations, are significant aspects of gene therapy development.1