HUMAN GENE-THERAPY

Current concepts in gene transfer and its application to the treatment of human genetic disorders, cancer, and other diseases are discussed. Gene therapy is a technique in which a functioning gene is inserted i nto a human cell to correct a genetic error or to introduce a new func tion to the cell. Many methods, including retroviral vectors, have bee n developed for ex vivo and in vivo gene insertion into cells. Some ph armacists have likened gene therapy to a sophisticated form of drug de livery and have envisioned an active role for the pharmacy profession. There are several safety and ethical issues related to manipulating t he human genome that need to be understood. Current gene therapy effor ts focus on gene insertion into somatic (non-germinal) cells only. Gen e therapy has the potential to revolutionize the treatment of genetic disorders, diseases associated with a genetic component (e.g., cystic fibrosis), cancer, AIDS, and many other diseases. Gene transfer may al so be used to better understand the biology of disease processes, such as the source of relapse in bone marrow transplant patients. The huma n genome project will undoubtedly lead to the identification, characte rization, and understanding of genes that are responsible for many hum an diseases, and gene therapy trials are sure to expand accordingly. T o date, over 40 clinical trials have been approved and more than 110 p atients have been entered in gene therapy studies. There are still man y technical obstacles to overcome before gene therapy can have widespr ead application. Injectable vectors need to be developed to simplify f oreign gene administration. Perhaps the biggest problem to overcome wi ll be engineering the target cells to be able to regulate gene express ion according to physiologic needs. Pharmacists should become knowledg eable about gene transfer techniques and possible clinical application s of gene therapy to keep abreast of the newest trends in medicine.