Duckydude1,
Below is a timeline of the progress of Gene Therapy, which is still in the infantile stages, yet can be used to treat certain genetic disorders (such as Alzheimer's). At this point, most geneticists are still trying to pinpoint which base pairs on which chromosomes are associated with which genes. It is a very large project, but if all scientists around the world work together, we can piece together everyone's discoveries to form the entire human genome. Even though we have already identified all the base pairs, we now have to figure out which ones work together to code for which genes. For example, eye color, may be a mix of "coding" from several base pairs on Chromosomes 8, 11, and 13 (just a ficticious example). At this point, we can't alter this, but who knows what the future holds. Currently, we use this information to know if certain proteins in our bodies are being over produced, under-produced, or not made at all. We then can "counter-act" those problems by giving shots of either more of that protien, or
antibodies that can bind to the excess protiens to excreet them from the system (if the body is making too much). See timeline below of current and past developments (based on Wikpedia):
2007
On 01 May 2007 Moorfields Eye Hospital and University College London's Institute of Opthalmology announced the world's first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23 year-old British male, Robert Johnson, in early 2007, but it is currently (31/05/2007) too early for results. [3]
2006
Scientists at the National Institutes of Health (Bethesda, Maryland) have successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. This study constitutes the first demonstration that gene therapy can be effective in treating cancer. The study results have been published in Science (October 2006).
In May 2006 a team of scientists led by Dr. Luigi Naldini and Dr. Brian Brown from the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) in Milan, Italy reported a breakthrough for gene therapy in which they developed a way to prevent the immune system from rejecting a newly delivered gene. Similar to organ transplantation, gene therapy has been plagued by the problem of immune rejection. So far, delivery of the 'normal' gene has been difficult because the immune system does not recognize the new gene and rejects the cells carrying it. To overcome this problem, the HSR-TIGET group utilized a newly uncovered network of genes regulated by molecules known as microRNAs. Dr. Naldini's group reasoned that they could use this natural function of microRNA to selectively turn off the identity of their therapeutic gene in cells of the immune system and prevent the gene from being found and destroyed. The researchers injected mice with the gene containing an immune-cell microRNA target sequence, and spectacularly, the mice did not reject the gene, as previously occurred when vectors without the microRNA target sequence were used. This work will have important implications for the treatment of hemophilia and other genetic diseases by gene therapy [4].
In March 2006 an international group of scientists announced the successful use of gene therapy to treat two adult patients for a disease affecting myeloid cells. The study, published in Nature Medicine, is believed to be the first to show that gene therapy can cure diseases of the myeloid system [5]
2003
In 2003 a University of California, Los Angeles research team inserted genes into the brain using liposomes coated in a polymer called polyethylene glycol (PEG). The transfer of genes into the brain is a significant achievement because viral vectors are too big to get across the "blood-brain barrier." This method has potential for treating Parkinson's disease. See Undercover genes slip into the brain at NewScientist.com (March 20, 2003).
RNA interference or gene silencing may be a new way to treat Huntington's. Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced. See Gene therapy may switch off Huntington's at NewScientist.com (March 13, 2003).
2002 and Earlier
New gene therapy approach repairs errors in messenger RNA derived from defective genes. Technique has potential to treat the blood disorder thalassaemia, cystic fibrosis, and some cancers. See Subtle gene therapy tackles blood disorder at NewScientist.com (October 11, 2002).
Researchers at Case Western Reserve University and Copernicus Therapeutics are able to create tiny liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane. See DNA nanoballs boost gene therapy at NewScientist.com (May 12, 2002).
Sickle cell is successfully treated in mice. See Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease from March 18, 2002, issue of The Scientist.
The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or "bubble boy" disease) held from 2000 and 2002 was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the United States, the United Kingdom, France, Italy, and Germany. (V. Cavazzana-Calvo, Thrasher and Mavilio 2004; see also 'Miracle' gene therapy trial halted at NewScientist.com, October 3, 2002).
In 1993 Andrew Gobea was born with a rare, normally fatal genetic disease - severe combined immunodeficiency (SCID). Genetic screening before birth showed that he had SCID. Blood was removed from Andre's placenta and umbilical cord immediately after birth, containing stem cells. The allele that codes for ADA was obtained and was inserted into a retrovirus. Retroviruses and stem cells were mixed, after which they entered and inserted the gene into the stem cells' chromosomes. Stem cells containing the working ADA gene were injected into Andre's blood system via a vein. For four years T-cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.
Problems and ethics
For the safety of gene therapy, the Weismann barrier is fundamental in the current thinking. Soma-to-germline feedback should therefore be impossible. However, there are indications [6] that the Weissman barrier can be breached. One way it might possibly be breached is if the treatment were somehow misapplied and spread to the testes and therefore would infect the germline against the intentions of the therapy.
Some of the problems of gene therapy include:
Short-lived nature of gene therapy - Before gene therapy can become a permanent cure for any condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be long-lived and stable. Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent gene therapy from achieving any long-term benefits. Patients will have to undergo multiple rounds of gene therapy.
Immune response - Anytime a foreign object is introduced into human tissues, the immune system is designed to attack the invader. The risk of stimulating the immune system in a way that reduces gene therapy effectiveness is always a potential risk. Furthermore, the immune system's enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients.
Problems with viral vectors - Viruses, while the carrier of choice in most gene therapy studies, present a variety of potential problems to the patient --toxicity, immune and inflammatory responses, and gene control and targeting issues. In addition, there is always the fear that the viral vector, once inside the patient, may recover its ability to cause disease.
Multigene disorders - Conditions or disorders that arise from mutations in a single gene are the best candidates for gene therapy. Unfortunately, some of the most commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of variations in many genes. Multigene or multifactorial disorders such as these would be especially difficult to treat effectively using gene therapy.
Chance of inducing a tumor (insertional mutagenesis) - If the DNA is integrated in the wrong place in the genome, for example in a tumor suppressor gene, it could induce a tumor (insertional mutagenesis). This has occurred in clinical trials for X-linked severe combined immunodeficiency (X-SCID) patients, in which hematopoietic stem cells were transduced with a corrective transgene using a retrovirus, and this led to the development of T cell leukemia in 3 of 20 patients.[1]
Deaths have occurred due to gene therapy, including that of Jesse Gelsinger.
from:http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
In popular culture
Gene therapy plays a major role in the sci-fi series Stargate Atlantis, as a certain type of alien technology can only be used if one has a certain gene which is given to the members of the team through gene therapy.
Gene therapy also plays a major role in the plot of the James Bond movie Die Another Day.
The Yellow Bastard from Frank Miller's Sin City was also apparently the recipient of gene therapy.
Gene therapy is a crucial plot element in the video game Metal Gear Solid, where it has been used to enhance the battle capabilities of enemy soldiers.
References
Durai, Sundar; Mala Mani, Karthikeyan Kandavelou, Joy Wu, Matthew H. Porteus, and Srinivasan Chandrasegaran (October 2005). "Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells". Nucleic Acids Research 33 (18): 59785990. DOI:10.1093/nar/gki912. PMID 16251401. Retrieved on 2006-05-28.
Gardlik, Roman; Roland Pálffy, Július Hodosy, Ján Lukács, Ján Turňa and Peter Celec (April 2005). "Vectors and delivery systems in gene therapy". Medical Science Monitor 11 (4): 110121. PMID 15795707. Retrieved on 2006-05-28.
Staff (November 18, 2005). Gene Therapy (FAQ). Human Genome Project Information. Oak Ridge National Laboratory. Retrieved on May 28, 2006.
Salmons B, Gunzburg WH (1993) "Targeting of retroviral vectors for gene therapy". Human Gene Therapy 4(2):129-141.
Baum C, Dullmann J, Li Z, Fehse B, Meyer J, Williams DA, von Kalle C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15;101(6):2099-114
Horn PA, Morris JC, Neff T, Kiem HP. Stem cell gene transfer--efficacy and safety in large animal studies. Molecular Therapy, 2004 Sep;10(3):417-31
Wang, Hongjie; Dmitry M. Shayakhmetov, Tobias Leege, Michael Harkey, Qiliang Li, Thalia Papayannopoulou, George Stamatoyannopolous, and André Lieber (September 2005). "A capsid-modified helper-dependent adenovirus vector containing the beta-globin locus control region displays a nonrandom integration pattern and allows stable, erythroid-specific gene expression". Journal of Virology 79 (17): 10999-11013. Retrieved on 2006-08-15.