Mark A. Kay
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Mark A. Kay
Mark A. Kay is an American physician-scientist recognized for his contributions to the field of gene therapy, genome editing, and RNA biology. He has an h-index of 123, and is known for liver-directed gene therapy, demonstrating RNA interference in mammals, and advancing adeno-associated virus (AAV) vector biology. Education Kay earned his B.S. in Physical Sciences from Lyman Briggs College at Michigan State University in 1980, and his M.D. and Ph.D. in Developmental Genetics from Case Western Reserve University in 1987. He completed a pediatric residency, medical genetics fellowship, and postdoctoral fellowship at Baylor College of Medicine in 1993. Career and research Kay began his work in gene therapy in 1990. He was a founding member of the American Society of Gene and Cell Therapy (ASGCT) in 1996 and served as its Vice President, President-Elect, and President from 2003 to 2006. He played a role in establishing the society’s journal, '' Molecular Therapy'', and advancing ...
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Gene Therapy
Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. 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. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in Phases of clinical research, phase I. In 2003, Gendicine became the first gene therapy to receive regulatory approval. Since that time, further gene therapy drugs were approved, such as alipogene tiparvovec (2012), Strimvelis (2016), tisagenlecleucel (2017), voretigene neparvovec ...
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Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical industry, pharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug interstate commerce, across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. Regulations are primarily at . Similar procedures are followed in the European Union, Japan, and Canada due to regulatory harmonization efforts by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, International Council for Harmonisation. Types * Research or investigator INDs are non-commercial INDs filed by researchers to study an unapproved drug or to study an approved drug for a new indication or in a new patient population. * Expanded access, Emergency Use INDs, also called compassionate use or single-patient INDs, are filed for emergency use of a ...
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H-index
The ''h''-index is an author-level metric that measures both the productivity and citation impact of the publications, initially used for an individual scientist or scholar. The ''h''-index correlates with success indicators such as winning the Nobel Prize, being accepted for research fellowships and holding positions at top universities. The index is based on the set of the scientist's most cited papers and the number of citations that they have received in other publications. The index has more recently been applied to the productivity and impact of a scholarly journal as well as a group of scientists, such as a department or university or country. The index was suggested in 2005 by Jorge E. Hirsch, a physicist at UC San Diego, as a tool for determining theoretical physicists' relative quality and is sometimes called the Hirsch index or Hirsch number. Hirsch intended the ''h''-index to address the main disadvantages of other bibliometric indicators. The total number of ...
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The New England Journal Of Medicine
''The New England Journal of Medicine'' (''NEJM'') is a weekly medical journal published by the Massachusetts Medical Society. Founded in 1812, the journal is among the most prestigious peer-reviewed medical journals. Its 2023 impact factor was 96.2, ranking it 2nd out of 168 journals in the category "Medicine, General & Internal". History In September 1811, Boston physician John Collins Warren, along with James Jackson, submitted a formal prospectus to establish the ''New England Journal of Medicine and Surgery and Collateral Branches of Science'' as a medical and philosophical journal. Subsequently, the first issue of the ''New England Journal of Medicine and Surgery and the Collateral Branches of Medical Science'' was published in January 1812. The journal was published quarterly. In 1823, another publication, the ''Boston Medical Intelligencer'', appeared under the editorship of Jerome V. C. Smith. The editors of the ''New England Journal of Medicine and Surgery and th ...
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Cell (journal)
''Cell'' is a peer-reviewed scientific journal publishing research papers across a broad range of disciplines within the life sciences. Areas covered include molecular biology, cell biology, systems biology, stem cells, developmental biology, genetics and genomics, proteomics, cancer research, immunology, neuroscience, structural biology, microbiology, virology, physiology, biophysics, and computational biology. The journal was established in 1974 by Benjamin LewinElsevier: ''Cell'': Home
(accessed 12 December 2008)
and is published twice monthly by Cell Press, owned by .


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Nature (journal)
''Nature'' is a British weekly scientific journal founded and based in London, England. As a multidisciplinary publication, ''Nature'' features Peer review, peer-reviewed research from a variety of academic disciplines, mainly in science and technology. It has core editorial offices across the United States, continental Europe, and Asia under the international scientific publishing company Springer Nature. ''Nature'' was one of the world's most cited scientific journals by the Science Edition of the 2022 ''Journal Citation Reports'' (with an ascribed impact factor of 50.5), making it one of the world's most-read and most prestigious academic journals. , it claimed an online readership of about three million unique readers per month. Founded in the autumn of 1869, ''Nature'' was first circulated by Norman Lockyer and Alexander MacMillan (publisher), Alexander MacMillan as a public forum for scientific innovations. The mid-20th century facilitated an editorial expansion for the j ...
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Business Wire
Business Wire is an American company that disseminates full-text press releases from thousands of companies and organizations worldwide to news media, financial markets, disclosure systems, investors, information web sites, databases, bloggers, social networks and other audiences. It is a wholly owned subsidiary of Berkshire Hathaway. History Business Wire was founded in 1961 by Lorry I. Lokey. It started by sending releases to 16 media outlets in California. Business Wire launched its website in May 1995. In 2000, ahead of its main competitor PR Newswire, Business Wire ended the practice of distributing news to financial outlets 15 minutes before anyone else, to provide immediate, equal access to company information as noted by the SEC's fair disclosure regulation ( Reg FD). Business Wire's first wholly owned European operation launched in 2001, with the opening of an office in London. On June 1, 2005, Business Wire entered the German Ad-Hoc market with a disclosure network ...
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Genetics
Genetics is the study of genes, genetic variation, and heredity in organisms.Hartl D, Jones E (2005) It is an important branch in biology because heredity is vital to organisms' evolution. Gregor Mendel, a Moravian Augustinians, Augustinian friar working in the 19th century in Brno, was the first to study genetics scientifically. Mendel studied "trait inheritance", patterns in the way traits are handed down from parents to offspring over time. He observed that organisms (pea plants) inherit traits by way of discrete "units of inheritance". This term, still used today, is a somewhat ambiguous definition of what is referred to as a gene. Phenotypic trait, Trait inheritance and Molecular genetics, molecular inheritance mechanisms of genes are still primary principles of genetics in the 21st century, but modern genetics has expanded to study the function and behavior of genes. Gene structure and function, variation, and distribution are studied within the context of the Cell (bi ...
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Pediatrics
Pediatrics (American English) also spelled paediatrics (British English), is the branch of medicine that involves the medical care of infants, children, Adolescence, adolescents, and young adults. In the United Kingdom, pediatrics covers many of their youth until the age of 18. The American Academy of Pediatrics recommends people seek pediatric care through the age of 21, but some pediatric subspecialists continue to care for adults up to 25. Worldwide age limits of pediatrics have been trending upward year after year. A physician, medical doctor who specializes in this area is known as a pediatrician, or paediatrician. The word ''pediatrics'' and its cognates mean "healer of children", derived from the two Ancient Greek, Greek words: (''pais'' "child") and (''iatros'' "doctor, healer"). Pediatricians work in clinics, research centers, universities, general hospitals and children's hospitals, including those who practice pediatric subspecialties (e.g. neonatology requires reso ...
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University Of Washington
The University of Washington (UW and informally U-Dub or U Dub) is a public research university in Seattle, Washington, United States. Founded in 1861, the University of Washington is one of the oldest universities on the West Coast of the United States. The university has a main campus located in the city's University District. It also has satellite campuses in nearby cities of Tacoma and Bothell. Overall, UW encompasses more than 500 buildings and over 20 million gross square footage of space, including one of the largest library systems in the world with more than 26 university libraries, art centers, museums, laboratories, lecture halls, and stadiums. Washington is the flagship institution of the six public universities in Washington State. It is known for its medical, engineering, and scientific research. Washington is a member of the Association of American Universities. According to the National Science Foundation, UW spent $1.73 billion on research and develo ...
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Springer Nature
Springer Nature or the Springer Nature Group is a German-British academic publishing company created by the May 2015 merger of Springer Science+Business Media and Holtzbrinck Publishing Group's Nature Publishing Group, Palgrave Macmillan, and Macmillan Education. History The company originates from several journals and publishing houses, notably Springer-Verlag, which was founded in 1842 by Julius Springer in Berlin (the grandfather of Bernhard Springer who founded Springer Publishing in 1950 in New York), Nature Portfolio, Nature Publishing Group which has published ''Nature (journal) , Nature'' since 1869, and Macmillan Education, which goes back to Macmillan Publishers founded in 1843. Springer Nature was formed in 2015 by the merger of Nature Publishing Group, Palgrave Macmillan, and Macmillan Education (held by Holtzbrinck Publishing Group) with Springer Science+Business Media (held by BC Partners). Plans for the merger were first announced on 15 January 2015. The transactio ...
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Cancer
Cancer is a group of diseases involving Cell growth#Disorders, abnormal cell growth with the potential to Invasion (cancer), invade or Metastasis, spread to other parts of the body. These contrast with benign tumors, which do not spread. Possible Signs and symptoms of cancer, signs and symptoms include a lump, abnormal bleeding, prolonged cough, unexplained weight loss, and a change in defecation, bowel movements. While these symptoms may indicate cancer, they can also have other causes. List of cancer types, 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 (nutrition), diet, sedentary lifestyle, lack of physical activity or Alcohol abuse, excessive alcohol consumption. Other factors include certain infections, exposure to ionizing radiation, and environmental pollutants. infectious causes of cancer, Infection with specific viruses, bacteria and parasites is an environmental factor cau ...
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