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Jesse Gelsinger
Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia – a byproduct of protein breakdown. The disease is usually fatal at birth, but Gelsinger had a milder form of the disease, in which the ornithine transcarbamylase gene is mutated in only part of the patient's cells, a condition known as somatic mosaicism. As his deficiency was partial, Gelsinger managed to survive on a restricted diet and special medications. Gelsinger joined a clinical trial run by the University of Pennsylvania that aimed at developing a treatment for infants born with the severe form of the disease. On September 13, 1999, Gelsinger was injected with an adenoviral vector carrying a corrected gene to test the safety of the procedure. He died four d ...
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Adenoviridae
Adenoviruses (members of the family ''Adenoviridae'') are medium-sized (90–100 nm), nonenveloped (without an outer lipid bilayer) viruses with an icosahedral nucleocapsid containing a double-stranded DNA genome. Their name derives from their initial isolation from human adenoids in 1953. They have a broad range of vertebrate hosts; in humans, more than 50 distinct adenoviral serotypes have been found to cause a wide range of illnesses, from mild respiratory infections in young children (known as the common cold) to life-threatening multi-organ disease in people with a weakened immune system. Virology Classification This family contains the following genera: * '' Atadenovirus'' * '' Aviadenovirus'' * '' Ichtadenovirus'' * '' Mastadenovirus'' (including all human adenoviruses) * '' Siadenovirus'' * '' Testadenovirus'' Diversity In humans, currently there are 88 human adenoviruses (HAdVs) in seven species (Human adenovirus A to G): * A: 12, 18, 31 * B: 3, 7, 11, 14, ...
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Clinical Trial Disasters
Clinical may refer to: Healthcare * Of or about a clinic, a healthcare facility * Of or about the practice of medicine Other uses * ''Clinical'' (film), a 2017 American horror thriller See also * * * Clinical chemistry, the analysis of bodily fluids for diagnostic and therapeutic purposes * Clinical death, the cessation of blood circulation and breathing * Clinical formulation, a theoretically-based explanation of information obtained from clinical assessment * Clinical governance, a systematic approach to maintaining and improving the quality of patient care * Clinical linguistics, linguistics applied to speech-language pathology * Clinical psychology, the understanding, preventing, and relieving psychologically-based distress or dysfunction * Clinical research, to determine the safety and effectiveness of medications etc. * Clinical significance, the practical importance of a treatment effect * Clinical trial, experiments or observations done in clinical research * Clinical wa ...
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1999 Deaths
File:1999 Events Collage.png, From left, clockwise: The funeral procession of King Hussein of Jordan in Amman; the 1999 İzmit earthquake kills over 17,000 people in Turkey; the Columbine High School massacre, one of the first major school shootings in the United States; the Year 2000 problem ("Y2K"), perceived as a major concern in the lead-up to the year 2000; the Millennium Dome opens in London; online music downloading platform Napster is launched, soon a source of online piracy; NASA loses both the Mars Climate Orbiter and the Mars Polar Lander; a destroyed T-55 tank near Prizren during the Kosovo War., 300x300px, thumb rect 0 0 200 200 Death and state funeral of King Hussein rect 200 0 400 200 1999 İzmit earthquake rect 400 0 600 200 Columbine High School massacre rect 0 200 300 400 Kosovo War rect 300 200 600 400 Year 2000 problem rect 0 400 200 600 Mars Climate Orbiter rect 200 400 400 600 Napster rect 400 400 600 600 Millennium Dome 1999 was designated as the ...
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1981 Births
Events January * January 1 ** Greece enters the European Economic Community, predecessor of the European Union. ** Palau becomes a self-governing territory. * January 10 – Salvadoran Civil War: The Farabundo Martí National Liberation Front, FMLN launches its first major offensive, gaining control of most of Morazán Department, Morazán and Chalatenango Department, Chalatenango departments. * January 15 – Pope John Paul II receives a delegation led by Polish Solidarity (Polish trade union), Solidarity leader Lech Wałęsa at the Vatican City, Vatican. * January 20 – Iran releases the 52 Americans held for 444 days, minutes after Ronald Reagan is First inauguration of Ronald Reagan, sworn in as the 40th President of the United States, ending the Iran hostage crisis. * January 21 – The first DMC DeLorean, DeLorean automobile, a stainless steel sports car with gull-wing doors, rolls off the production line in Dunmurry, Northern Ireland. * January 24 – An 1981 Dawu ea ...
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University Of Utah
The University of Utah (U of U, UofU, or simply The U) is a public research university in Salt Lake City, Utah. It is the flagship institution of the Utah System of Higher Education. The university was established in 1850 as the University of Deseret () by the General Assembly of the provisional State of Deseret, making it Utah's oldest institution of higher education. It received its current name in 1892, four years before Utah attained statehood, and moved to its current location in 1900. As of Fall 2019, there were 24,485 undergraduate students and 8,333 graduate students, for an enrollment total of 32,818, making it the second largest public university in the state after Utah Valley University. Graduate studies include the S.J. Quinney College of Law and the School of Medicine, Utah's first medical school. It is a member of the Association of American Universities (AAU) and is classified among "R1: Doctoral Universities – Very high research activity". According to the ...
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Side Effect
In medicine, a side effect is an effect, whether therapeutic or adverse, that is secondary to the one intended; although the term is predominantly employed to describe adverse effects, it can also apply to beneficial, but unintended, consequences of the use of a drug. Developing drugs is a complicated process, because no two people are exactly the same, so even drugs that have virtually no side effects, might be difficult for some people. Also, it is difficult to make a drug that targets one part of the body but that does not affect other parts, the fact that increases the risk of side effects in the untargeted parts. Occasionally, drugs are prescribed or procedures performed specifically for their side effects; in that case, said side effect ceases to be a side effect and is now an intended effect. For instance, X-rays were historically (and are currently) used as an imaging technique; the discovery of their oncolytic capability led to their employ in radiotherapy (ablation o ...
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James Wilson (scientist)
James M. Wilson is a biomedical researcher with expertise in gene therapy. Wilson graduated from Albion College (B.A., Chemistry) and the University of Michigan (MD, PhD).  He completed residency training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute. Wilson serves as the Director of the Gene Therapy Program, Rose H. Weiss Professor and Director of the Orphan Disease Center, and Professor of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. Previously, he held the John Herr Musser endowed professorship at the Perelman School of Medicine. Wilson led a clinical trial that resulted in the death of Jesse Gelsinger. As a result, the government banned him from working on FDA-regulated human clinical trials for five years, which led to a shift in his research focus towards a study of adeno-associated viruses (AAV). Scientific contributions Wilson’s research invo ...
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Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C), but the agency also enforces other laws, notably Section 361 of the Public Health Service Act, as well as associated regulations. Much of this regulatory-enforcement work is not d ...
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University Of Pennsylvania
The University of Pennsylvania (also known as Penn or UPenn) is a private research university in Philadelphia. It is the fourth-oldest institution of higher education in the United States and is ranked among the highest-regarded universities by numerous organizations and scholars. While the university dates its founding to 1740, it was created by Benjamin Franklin and other Philadelphia citizens in 1749. It is a member of the Ivy League. The university has four undergraduate schools as well as twelve graduate and professional schools. Schools enrolling undergraduates include the College of Arts and Sciences, the School of Engineering and Applied Science, the Wharton School, and the School of Nursing. Among its highly ranked graduate schools are its law school, whose first professor wrote the first draft of the United States Constitution, its medical school, the first in North America, and Wharton, the first collegiate business school. Penn's endowment is US$20.7 billio ...
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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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Somatic Mosaicism
Mosaicism or genetic mosaicism is a condition in multicellular organisms in which a single organism possesses more than one genetic line as the result of genetic mutation. This means that various genetic lines resulted from a single fertilized egg. Genetic mosaics may often be confused with chimerism, in which two or more genotypes arise in one individual similarly to mosaicism. In chimerism, though, the two genotypes arise from the fusion of more than one fertilized zygote in the early stages of embryonic development, rather than from a mutation or chromosome loss. Genetic mosaicism can result from many different mechanisms including chromosome nondisjunction, anaphase lag, and endoreplication. Anaphase lagging is the most common way by which mosaicism arises in the preimplantation embryo. Mosaicism can also result from a mutation in one cell during development, in which case the mutation will be passed on only to its daughter cells (and will be present only in certain adult cel ...
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