Human gene therapy is the treatment of human diseases with deoxyribonucleic acid fragments comprising whole genes or parts of genes.
Keywords: gene; disease; viral; nonviral; therapy
Human Gene Therapy
Panos A Ioannou, University of Melbourne, Royal Children's Hospital, Parkville, Victoria, Australia
Published online: January 2006
DOI: 10.1038/npg.els.0003841
| Further Reading | |
| Baum C, Dullmann J, Li Z et al. (2003) Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 101: 2099–2114 (Review). | |
| Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669–672. | |
| Evans JT and Garcia JV (2000) Lentivirus vector mobilization and spread by human immunodeficiency virus. Human Gene Therapy 11: 2331–2339. | |
| Felgner PL (1997) Nonviral strategies for gene therapy. Scientific American 276: 102–106. | |
| Fox JL (2000) Gene-therapy death prompts broad civil lawsuit. Nature Biotechnology 18: 1136. | |
| Friedmann T (1997) Overcoming the obstacles to gene therapy. Scientific American 276: 96–101. | |
| Friend SH (1999) How DNA microarrays and expression profiling will affect clinical practice. British Medical Journal 319: 1306. | |
| book Kresina TF (ed.) (2001) An Introduction to Molecular Medicine and Gene Therapy. New York: Wiley-Liss. | |
| Michael A (1996) Financing gene therapy beyond phase II. Gene Therapy 3: 1035–1038. | |
| Morgan RA and Blaese RM (1999) Gene therapy: lessons learned from the past decade. British Medical Journal 319: 1310. | |
| Orford M, Nefedov M, Vadolas J et al. (2000) Engineering EGFP reporter constructs into a 200 kb human globin BAC clone using GET recombination. Nucleic Acids Research 28(84): 1–8. | |
| Penn SG, Rank DR, Hanzel DK and Barker DL (2000) Mining the human genome using microarrays of open reading frames. Nature Genetics 26: 315–318. | |
| Thompson L (2000) Human gene therapy. Harsh lessons, high hopes. FDA Consumer 34(5) [http://www.fda.gov/fdac/features/2000/500_gene.html]. | |
| book Walther W and Stein U (eds) (2000) Gene Therapy of Cancer: Methods and Protocols. Totowa, NJ: Humana Press. | |
| Zhao S (2001) A comprehensive BAC resource. Nucleic Acids Research 29: 141–143. | |
