Jess,+Alyce,+Emily,+Liam+-+Gene+Therapy

=//Liam Samat, Jess Ellisdon, Alyce Williams and Emily Faulkner's page on "GENE THERAPY"// =

toc

 **//__Definitions__//**

**All words in bold appear in the definitions section.**

**What is Gene Therapy?**
Gene Therapy uses genes as medicine. Surgeons take the gene and transfers a working gene into a specific cell, so that the faulty gene is repaired. Thus the gene can replace a gene that is no working. Gene Therapy can also be use to introduce a new gene into the body, which can overwrite a cancer gene. The falty gene never goes away but the introduced gene over powers the old one.

**Gene Therapy cons:**

 * Our immune system immediately attacks any foreign object that is inserted into the human body, so there is a risk that our immune system will attack the gene that is inserted.
 * If everyone with a disease or genetic complication received gene therapy, the world would over populate.
 * The gene added isn't always successful in the first round you may have to undergo several frequent treatments.
 * It could be used to genetically enhance humans to an unfair advantage.

**Gene Therapy pros:**

 * Gene therapy will be a wonderful thing for serious diseases.
 * You would save loved ones.

**Ethical opinions: **
Technological questions in this area are being answered rapidly. However, the ethical questions that have appeared as a result of technology are not being answered quite so rapidly. Until the development of this technology, people have had to deal with genetic inequality as a fact of life. With the advent of gene therapy, this may no longer be the case for some people. Many people feel it is alright to use gene therapy to treat human genetic diseases. Surprisingly, even the Catholic Church has approved for the use of gene therapy. Reverend Russell E. Smith, president of the Pope John XXIII Medical-Moral Research and Education Center, stated that gene therapy is "a very noble enterprise, because it is aimed at the actual cure of actual diseases." Individuals, are concerned that the technique may be being used for "treatment" of genetic "disorders" rather than other diseases.

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;"><range type="comment" id="920925">For example, in January of 1993, it was reported in USA Today that an 11 year old boy was receiving gene therapy treatments at a cost of $150,000 per year to increase his height. At 4' 11", four inches below average height, he was tired of being picked on at school for being short. His father was quoted in the article as saying, "You want to give your child that edge no matter what. I think you'd do just about anything." <span style="color: #0000ff; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 90%;">Taken from: <span style="color: #0000ff; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 90%;">[]

<span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**How is Gene Therapy delivered?**
<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">The problem surgeons are facing is finding a way to deliver the working gene into the body. At the start, the cells that are affected are removed from the persons body. Then a working copy of the gene is ** SPLICED ** or injected into the removed cells. These cells are left to grow in a laboratory, then replaced into the person they were removed from. Another promising technique is to have the working copy of the cell placed inside a very harmless virus, that has had most of the genes inside it removed-this means it has been deactivated.

<span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**Examples of Gene Therapy:**

 * 1) <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">In February 2007, scientists trialed gene therapy at the NIHR Bio-medical Research centre in the US, conducting the experiments on three patients. The patients were around 18 years of age. The scientists were trialing the therapy on a condition called "Leber's congenital amaurosis" or LCA

<span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**Success stories:**

 * 1) <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Last year in France, scientist tried gene therapy on 3 children that suffered from SCID (Severe Immuno Deficiency Disorder), in which cells of the immune system don't function-were treated with gene therapy. The three boys now have a normal immune cell function after receiving injection of normal genes into their immune cells.

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">To see more success stories and other information follow the links <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[]

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;"> **Unsuccessful** ** stories: **
<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">1. In 1999 Gene Therapy was setback when an 18 year old Jesse Gelsinger died after a trail of gene therapy for OCTD, <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">(Ornithine Transcarpoxylase Defiency). <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">He died 4 days later from organ failure. <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">His death is thought to have been caused by a severe immune response in his body.

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">** What diseases is gene therapy available for? **
<span style="color: #000000; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">gene therapy is being tested to treat over 1000 diseases some include:
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Hemophilia
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Some cancers
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Sever combined immune deficiency
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Cystic fibrosis
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Alzheimer's disease

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[]
 * <span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Australian Government's Opinion: **

<span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**Newspaper Articles:**
<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 130%;">Gene therapy for tot leads to leukemia <span style="background-color: #ffffff; color: #333333; display: block; font-family: Arial; font-size: 14px; text-align: left;"><span style="background-color: #ffffff; color: #000000; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">LONDON: One of the first children in Britain to receive gene therapy for an immune system disorder has developed leukemia as a result of his treatment. The boy, aged 3, is the first gene therapy patient in Britain to fall ill with leukemia, a known risk of the procedure. A similar program in France has caused four cases of the blood cancer and one death. The child, who has not been named, was born with X-linked severe combined immunodeficiency (X-SCID), a condition in which the immune system fails to develop. It is often called "bubble baby syndrome" because sufferers are shielded in a sterile pouch. Without treatment, they die in their first year from infections such as pneumonia or chicken pox. Two years ago, the boy became the eighth patient to be treated in London on Great Ormond Street Hospital's gene therapy program, which uses a genetically modified virus to correct the faulty DNA that causes X-SCID. While his immune system responded well to the procedure, leukemia was diagnosed last month, the hospital said. This is an acknowledged risk of gene therapy, because inserting the replacement DNA can activate another gene that promotes cancer. None of the other 14 British children who have had gene therapy for X-SCID and a similar condition, known as ada-SCID, has developed leukemia so far, while all have had immune function restored. The cancer, however, has been diagnosed in four of the 11 patients involved in a French trial, one of whom has died while three are in remission. While 80 per cent of children with leukemia make a full recovery, untreated X-SCID is always fatal. The doctors said that gene therapy was usually a better option than the alternative - a bone marrow transplant. A suitable bone marrow donor is available for only a third of X-SCID children, and even when a transplant is possible it carries a risk of complications, including death, because the children are so ill. Doctors said there was every chance that the boy would survive leukemia and be cured of X-SCID. The trial has been closed to new patients but another program using a slightly different technique will start next year. Martin Gore, chairman of the Government's Gene Therapy Advisory Committee, said the data his committee had seen "suggests that the risk of leukemia after gene therapy may be confined to this patient group."-FROM THE AUSTRALIAN
 * <span style="color: #277f9c; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 11px;"> BY: MARK HENDERSON, LONDON
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;"> From: [|The Australian]
 * <span style="color: #918f75; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 11px;"> December 20, 2007 12:00AM

<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 130%;">Gene cure for blindness <span style="background-color: #ffffff; color: #333333; display: block; font-family: Arial; font-size: 14px; text-align: left;"> <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**A BRITISH hospital has made the world's <span style="color: #000000; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 115%;">first attempt t o treat blindness with a revolutionary gene therapy.** Surgeons at the Moorfields Eye Hospital in London operated on Robert Johnson, who was born with a rare sight disorder known as Leber’s congenital amaurosis (LCA), which deteriorates with age. Mr Johnson, 23, who had genes inserted into one eye, could see only outlines during the day and very little at night before having the procedure yesterday. He is one of a dozen young patients selected for the first clinical trial to test the new therapy, which has already proved successful at restoring the sight of dogs in tests. It will be months before the researchers know whether their work has been a success, but it is thought that the therapy could be used to treat a wide range of inherited sight disorders in adults and children. <span style="background-color: #ffffff; color: #333333; display: block; font-family: Arial; font-size: 14px; text-align: left;"><span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">The LCA disorder is caused by a defect in a gene called RPE65, which prevents the light-sensitive layer of cells in the retina at the back of the eye from working properly. Usually these are cells that detect light, but in Mr Johnson’s case they are damaged and prevent him from seeing properly. The operation, conceived by researchers from University College London, involved injecting working copies of the defective gene into the back of the eye. Surgeons used a harmless virus or “vector” to carry the gene into the cells. It is hoped that the replacement genes will enable the retina to detect light – and eventually restore Mr Johnson’s sight. The trial, funded by the Department of Health, involves 12 adults and children with LCA, for which there are currently no effective treatments. During preliminary studies, the vision of dogs with the defect was restored to the extent that they were able to walk through a maze without difficulty; something they could not do before the treatment. The purpose of the Moorfields trial is to find out how safe and effective the intervention is for humans. The researchers hope that their work could lead to ways to treat more common sight problems, such as age-related macular degeneration, which affects about 250,000 Britons. Most previous gene therapies have been developed in an attempt to treat different types of cancer. Before surgery, Mr Johnson told the BBC that he had mixed feelings. He said: “It’s very difficult to say how I’m feeling. I keep ranging from extreme nervousness to a bit of excitement.” Professor Robin Ali, the lead researcher, based at the Institute of Ophthalmology, has spent 15 years working with colleagues developing the technique. He said yesterday: “I can’t help feeling somewhat apprehensive. There is so much riding on it and we have all been waiting for a very long time.” His colleague, James Bainbridge, who carried out the surgery, said that there was no guarantee that it would be a success. However, he added: “It is very encouraging that we can deliver genes to an extremely fragile site in the eye without complications.” The surgery required incredible precision. Robert Maclaren, the assistant surgeon, said yesterday that he was pleased with how things went. “We couldn’t have asked for a better result,” he said. Professor Ali added: “There are many forms of retinal degeneration, meaning the use of gene therapy treatments must be individually developed, then tested in a separate clinical trial specifically for that disease. “However, the results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future.”-FROM THE TIMES <span style="background-color: #ffffff; color: #333333; display: block; font-family: Arial; font-size: 14px; text-align: left;"> **<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">THREE US cancer patients were brought back from the brink by a new therapy that turned their own immune cells into tumour killers, wiping out an advanced form of <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: medium; line-height: 27px;">leukemia <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">, researchers say. ** <span style="background-color: #ffffff; color: #333333; font-family: Arial; font-size: 14px; text-align: left;"><span style="color: #000000; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">The breakthrough stunned scientists and although the gene transfer therapy technique is still in development, it could offer hope one day to people who suffer from ovarian, lung, breast and skin cancers. "We saw amazing results," said Michael Kalos, lead author of the study that appeared in Science Translational Medicine and was published simultaneously in the New England Journal of Medicine. "These were nasty tumours that were late-stage, a lot of mutations that had bad prognosis," he said. "We saw massive reduction in tumour burden. One patient had over seven pounds (three kilograms) of tumour and it all disappeared." Two of the three men in the study with chronic lymphocytic leukaemia (CLL) have remained cancer-free for almost a year, while the third has seen a slight recurrence of disease.
 * <span style="color: #277f9c; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 11px;"> BY: DAVID ROSE
 * <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;"> From: The Times
 * <span style="color: #918f75; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif; font-size: 11px;"> May 02, 2007 12:00AM

<span style="background-color: #ffffff; color: #333333; font-family: Arial; font-size: 14px; text-align: left;"><span style="color: #000000; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">Within three weeks, the tumours had been blown away, in a way that was much more violent than we ever expected," said senior author Carl June, who like Kalos is a researcher at the University of Pennsylvania. "It worked much better than we thought it would." Scientists removed a sample of the patients' T-cells and genetically modified them to attack all cells that express a certain kind of protein, CD19, which includes tumour cells. They altered them using a lentivirus vector that encodes an antibody-like protein known as a chimeric antigen receptor. The protein is expressed on the surface of T-cells and designed to bind to CD19. The scientists also engineered the T-cells to start triggering other T-cells to multiply as soon as they attached to a cancer cell, bringing on a faster death for the tumour but avoiding the side-effects of cancer drugs. "We saw at least a 1000-fold increase in the number of modified T-cells in each of the patients. Drugs don't do that," said June, describing the infused T-cells as "serial killers". "On average, each infused T-cell led to the killing of thousands of tumour cells." In one case, a 64-year-old man had blood and marrow "replete with tumour cells". He saw little change for the first two weeks after treatment, but then started experiencing nausea, chills and fever. Tests showed he was undergoing a huge rise in T-cell count, and a condition known as tumour lysis syndrome that can arise when cancer cells are dying off. By day 28, his blood showed no evidence of leukaemia. A 65-year-old patient saw similar results, with no trace of leukaemia after a year, but a 77-year-old patient saw a slight recurrence of cancer after he was treated with steroids for the symptoms of tumour lysis syndrome. However, his tumour load remains far below what it was before the treatment. Steven Rosenberg, chief of the surgery branch at the National Cancer Institute, described it as "important" and "impressive". He was not involved with the study but has published research on similar approaches to eradicating B-cell lymphomas and melanoma. "You are taking advantage of the body's immune system by creating outside the body T-cells that can act against the cancer," he told AFP. "You have to select the particular gene modification for each type of cancer but when you pick it wisely it can be very effective." The other main form of treatment, bone marrow transplants, carries a minimum 20 per cent risk of dying from the procedure and cure rates hover at around 50 per cent. While it remains unknown how long the treatment may keep cancer at bay, researchers were excited to see that "memory" T-cells remained months after the cancer disappeared, indicating the body is retaining some protection. The next step is to try the technique in two children and at least 13 adults with CD19-positive leukaemia. They are also looking to determine whether the approach could target non-Hodgkin's lymphoma and acute lymphocytic leukaemia, mesothelioma cancer cells, ovarian and pancreatic cancer cells. Chronic lymphocytic leukaemia is the second most common type of adult leukemia after acute myeloid leukemia, according to the National Cancer Institute. "I'm healthy and still in remission," said a statement by one of the three patients, who declined to be named. "I know this may not be a permanent condition, but I decided to declare victory."

<span style="color: #572ab7; font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">**Bibliography/References:**
<span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[|http://www.genetics.edu.au/pdf/factsheets/fs27.pdf] <span style="font-family: 'Lucida Sans Unicode','Lucida Grande',sans-serif;">[]