2002 French Gene Therapy Trials
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2002 French Gene Therapy Trials
The 2002 French gene therapy trials were experimental gene therapy trials performed on children suffering from severe combined immunodeficiency (SCID). The trials were based in Paris and led by researchers Alain Fischer and Marina Cavazzana-Calvo. Whilst the experiment initially seemed successful, many of the children began showing symptoms of various cancer-like diseases as a result of the gene manipulation. The experiment, and others like it, were subsequently shut down. Trials The trials took place at the Necker Hospital in Paris, France in 2002. The lead researchers were Dr. Alain Fischer and Dr. Marina Cavazzana-Calvo, who were both employed by the hospital. The researchers were investigating treatments for severe combined immune deficiency (SCID), a disease that had been linked to the X-chromosome. SCID has the effect of preventing the formation of several key immune system factors that aid in the body's ability to fight of infectious diseases. The goal of the gene therapy w ...
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Gene Therapy
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.Gene Therapy Cli ...
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Severe Combined Immunodeficiency
Severe combined immunodeficiency (SCID), also known as Swiss-type agammaglobulinemia, is a rare genetic disorder characterized by the disturbed development of functional T cells and B cells caused by numerous genetic mutations that result in differing clinical presentations. SCID involves defective antibody response due to either direct involvement with B lymphocytes or through improper B lymphocyte activation due to non-functional T-helper cells. Consequently, both "arms" (B cells and T cells) of the adaptive immune system are impaired due to a defect in one of several possible genes. SCID is the most severe form of primary immunodeficiencies, and there are now at least nine different known genes in which mutations lead to a form of SCID. It is also known as the bubble boy disease and bubble baby disease because its victims are extremely vulnerable to infectious diseases and some of them, such as David Vetter, have become famous for living in a sterile environment. SCID is the ...
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Paris
Paris () is the capital and most populous city of France, with an estimated population of 2,165,423 residents in 2019 in an area of more than 105 km² (41 sq mi), making it the 30th most densely populated city in the world in 2020. Since the 17th century, Paris has been one of the world's major centres of finance, diplomacy, commerce, fashion, gastronomy, and science. For its leading role in the arts and sciences, as well as its very early system of street lighting, in the 19th century it became known as "the City of Light". Like London, prior to the Second World War, it was also sometimes called the capital of the world. The City of Paris is the centre of the Île-de-France region, or Paris Region, with an estimated population of 12,262,544 in 2019, or about 19% of the population of France, making the region France's primate city. The Paris Region had a GDP of €739 billion ($743 billion) in 2019, which is the highest in Europe. According to the Economist Intelli ...
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Marina Cavazzana
Marina Cavazzana is a professor of Paediatric Immunology at the Necker-Enfants Malades Hospital and the Imagine Institute, as well as an academic at Paris Descartes University. She was awarded the Irène Joliot-Curie Prize in 2012 and elected to the National Academy of Medicine in 2019. Early life and education Cavazzana was born in Venice. Her father worked on the railway and her mother was a teacher. She studied medicine in Padua, where she completed her residency at the University of Padua. She moved to Paris, where she earned her doctorate at the Paris Diderot University studying bone marrow transplants with Élaine Gluckman at the Hôpital Saint-Louis. Research and career Cavazzana joined the Necker-Enfants Malades Hospital in 1987, where she worked with Alain Fischer and Claude Gricelli. She was appointed to Professor of Immunology in 1994. She served as Director of the Institut national de la santé et de la recherche médicale (INSERM) in 1994. In 2003 she was ma ...
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Cancer
Cancer is a group of diseases involving abnormal cell growth with the potential to invade or spread to other parts of the body. These contrast with benign tumors, which do not spread. Possible signs and symptoms include a lump, abnormal bleeding, prolonged cough, unexplained weight loss, and a change in bowel movements. While these symptoms may indicate cancer, they can also have other causes. Over 100 types of cancers affect humans. Tobacco use is the cause of about 22% of cancer deaths. Another 10% are due to obesity, poor diet, lack of physical activity or excessive drinking of alcohol. Other factors include certain infections, exposure to ionizing radiation, and environmental pollutants. In the developing world, 15% of cancers are due to infections such as ''Helicobacter pylori'', hepatitis B, hepatitis C, human papillomavirus infection, Epstein–Barr virus and human immunodeficiency virus (HIV). These factors act, at least partly, by changing the genes of ...
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Leukemia
Leukemia ( also spelled leukaemia and pronounced ) is a group of blood cancers that usually begin in the bone marrow and result in high numbers of abnormal blood cells. These blood cells are not fully developed and are called ''blasts'' or ''leukemia cells''. Symptoms may include bleeding and bruising, bone pain, fatigue, fever, and an increased risk of infections. These symptoms occur due to a lack of normal blood cells. Diagnosis is typically made by blood tests or bone marrow biopsy. The exact cause of leukemia is unknown. A combination of genetic factors and environmental (non-inherited) factors are believed to play a role. Risk factors include smoking, ionizing radiation, petrochemicals (such as benzene), prior chemotherapy, and Down syndrome. People with a family history of leukemia are also at higher risk. There are four main types of leukemia— acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL) and chronic myeloi ...
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Gene Therapy
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.Gene Therapy Cli ...
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2002 In Science
The year 2002 in science and technology involved some significant events Astronomy and space science * February 19 – NASA's ''2001 Mars Odyssey'' space probe begins to map the surface of Mars using its thermal emission imaging system. * May 26 – The ''Mars Odyssey'' finds signs of huge water ice deposits on the planet Mars. * June 4 – Quaoar is discovered. * June 6 – 2002 Eastern Mediterranean event: An object with an estimated diameter of 10 meters impacts with Earth's atmosphere over the Mediterranean Sea and detonates in mid-air. * June 10 – Solar eclipse of June 10, 2002: Annular solar eclipse. * September 25 – 2002 Vitim event: Possible bolide impact in Irkutsk Oblast, Russia. * December 4 – Solar eclipse of December 4, 2002: Total solar eclipse. Biology * 18 April – New suborder of insects, Mantophasmatidae, announced. * Publication of ''Systema Porifera: a guide to the classification of sponges'' edited by John N. A. Hooper and Rob W. M. Van Soest. Carto ...
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2002 In France
Events from the year 2002 in France. Incumbents * President: Jacques Chirac * Prime Minister: Lionel Jospin (until 7 May), Jean-Pierre Raffarin (starting 7 May) Events *1 January – the Euro replaces the French franc as France's currency. *6 March – France agrees to return the remains of Saartjie Baartman to South Africa. *21 April – first round of the presidential election: Jacques Chirac and Jean-Marie le Pen poll the most votes. Two weeks of demonstrations against the National Front follow. *5 May – second round of the presidential election. Jacques Chirac returns for a second term as President of the French Republic. *6 May – nomination of Jean-Pierre Raffarin for the post of Prime Minister. *9 June – first round of the legislative elections *16 June – second round of legislative elections. *14 July – During Bastille Day celebrations, Jacques Chirac escapes an assassination attempt unscathed. *November – Citroën launches a revised model of its popular Be ...
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