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Alipogene Tiparvovec
Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis. It was recommended for approval by the European Medicines Agency in July 2012, and approved by the European Commission in November of the same year. It was the first marketing authorisation for a gene therapy treatment in either the European Union or the United States. The medication is administered via a series of injections into the leg muscles. Glybera gained infamy as the "million-dollar drug" and proved commercially unsuccessful for a number of reasons. Its cost to patients and payers, together with the rarity of LPLD, high maintenance costs to its manufacturer , and failure to achieve approval in the US, led to withdrawing the drug after two years on the EU market. As of 2018, only 31 people worldwide have ever been administered Glybera, and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lipoprotein Lipase
Lipoprotein lipase (LPL) (EC 3.1.1.34, systematic name triacylglycerol acylhydrolase (lipoprotein-dependent)) is a member of the lipase gene family, which includes pancreatic lipase, hepatic lipase, and endothelial lipase. It is a water-soluble enzyme that hydrolyzes triglycerides in lipoproteins, such as those found in chylomicrons and very low-density lipoproteins (VLDL), into two free fatty acids and one monoacylglycerol molecule: : triacylglycerol + H2O = diacylglycerol + a carboxylate It is also involved in promoting the cellular uptake of chylomicron remnants, cholesterol-rich lipoproteins, and free fatty acids. LPL requires ApoC-II as a cofactor. LPL is attached to the luminal surface of endothelial cells in capillaries by the protein glycosylphosphatidylinositol HDL-binding protein 1 ( GPIHBP1) and by heparan sulfated peptidoglycans. It is most widely distributed in adipose, heart, and skeletal muscle tissue, as well as in lactating mammary glands. Synthesis ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Quebec
Quebec is Canada's List of Canadian provinces and territories by area, largest province by area. Located in Central Canada, the province shares borders with the provinces of Ontario to the west, Newfoundland and Labrador to the northeast, New Brunswick to the southeast and a coastal border with the territory of Nunavut. In the south, it shares a border with the United States. Between 1534 and 1763, what is now Quebec was the List of French possessions and colonies, French colony of ''Canada (New France), Canada'' and was the most developed colony in New France. Following the Seven Years' War, ''Canada'' became a Territorial evolution of the British Empire#List of territories that were once a part of the British Empire, British colony, first as the Province of Quebec (1763–1791), Province of Quebec (1763–1791), then Lower Canada (1791–1841), and lastly part of the Province of Canada (1841–1867) as a result of the Lower Canada Rebellion. It was Canadian Confederation, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antibody-dependent Cellular Cytotoxicity
Antibody-dependent cellular cytotoxicity (ADCC), also referred to as antibody-dependent cell-mediated cytotoxicity, is a mechanism of cell-mediated immune defense whereby an effector cell of the immune system kills a target cell, whose membrane-surface antigens have been bound by specific antibodies. It is one of the mechanisms through which antibodies, as part of the humoral immune response, can act to limit and contain infection. ADCC is independent of the immune complement system that also lyses targets but does not require any other cell. ADCC requires an effector cell which classically is known to be natural killer (NK) cells that typically interact with immunoglobulin G (IgG) antibodies. However, macrophages, neutrophils and eosinophils can also mediate ADCC, such as eosinophils killing certain parasitic worms known as helminths via IgE antibodies. In general, ADCC has typically been described as the immune response to antibody-coated cells leading ultimately to the l ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antibodies
An antibody (Ab) or immunoglobulin (Ig) is a large, Y-shaped protein belonging to the immunoglobulin superfamily which is used by the immune system to identify and neutralize antigens such as bacteria and viruses, including those that cause disease. Each individual antibody recognizes one or more specific antigens, and antigens of virtually any size and chemical composition can be recognized. Antigen literally means "antibody generator", as it is the presence of an antigen that drives the formation of an antigen-specific antibody. Each of the branching chains comprising the "Y" of an antibody contains a paratope that specifically binds to one particular epitope on an antigen, allowing the two molecules to bind together with precision. Using this mechanism, antibodies can effectively "tag" the antigen (or a microbe or an infected cell bearing such an antigen) for attack by cells of the immune system, or can neutralize it directly (for example, by blocking a part of a viru ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunogenicity
Immunogenicity is the ability of a foreign substance, such as an antigen, to provoke an immune response in the body of a human or other animal. It may be wanted or unwanted: * Wanted immunogenicity typically relates to vaccines, where the injection of an antigen (the vaccine) provokes an immune response against the pathogen, protecting the organism from future exposure. Immunogenicity is a central aspect of vaccine development. * Unwanted immunogenicity is an immune response by an organism against a therapeutic antigen. This reaction leads to production of anti-drug-antibodies (ADAs), inactivating the therapeutic effects of the treatment and potentially inducing adverse effects. A challenge in biotherapy is predicting the immunogenic potential of novel protein therapeutics. For example, immunogenicity data from high-income countries are not always transferable to low-income and middle-income countries. Another challenge is considering how the immunogenicity of vaccines changes wi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Retrovirus
A retrovirus is a type of virus that inserts a DNA copy of its RNA genome into the DNA of a host cell that it invades, thus changing the genome of that cell. After invading a host cell's cytoplasm, the virus uses its own reverse transcriptase enzyme to produce DNA from its RNA genome, the reverse of the usual pattern, thus ''retro'' (backward). The new DNA is then retroviral integration, incorporated into the host cell genome by an integrase enzyme, at which point the retroviral DNA is referred to as a provirus. The host cell then treats the viral DNA as part of its own genome, transcribing and translating the viral genes along with the cell's own genes, producing the proteins required to assemble new copies of the virus. Many retroviruses cause serious diseases in humans, other mammals, and birds. Retroviruses have many subfamilies in three basic groups. * Oncovirus, Oncoretroviruses (cancer-causing retroviruses) include human T-lymphotropic virus (HTLV) causing a type of leuk ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunosuppressive Therapy
Immunosuppression is a reduction of the activation or efficacy of the immune system. Some portions of the immune system itself have immunosuppressive effects on other parts of the immune system, and immunosuppression may occur as an adverse reaction to treatment of other conditions. In general, deliberately induced immunosuppression is performed to prevent the body from rejecting an organ transplant. Additionally, it is used for treating graft-versus-host disease after a bone marrow transplant, or for the treatment of auto-immune diseases such as systemic lupus erythematosus, rheumatoid arthritis, Sjögren's syndrome, or Crohn's disease. This is typically done using medications, but may involve surgery (splenectomy), plasmapheresis, or radiation. A person who is undergoing immunosuppression, or whose immune system is weak for some other reasons (such as chemotherapy or HIV), is said to be ''immunocompromised''. Deliberately induced Administration of immunosuppressive medica ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adeno-associated Virus
Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus '' Dependoparvovirus'', which in turn belongs to the family ''Parvoviridae''. They are small (approximately 26 nm in diameter) replication-defective, nonenveloped viruses and have linear single-stranded DNA (ssDNA) genome of approximately 4.8 kilobases (kb). Several features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell. In the native virus, however, integration of virally carried genes into the host genome does occur. Integration can be important for certain applications, but can also have unwanted consequences. Recent human clinical trials using AAV for gene therapy in the retina have shown ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Italy
Italy, officially the Italian Republic, is a country in Southern Europe, Southern and Western Europe, Western Europe. It consists of Italian Peninsula, a peninsula that extends into the Mediterranean Sea, with the Alps on its northern land border, as well as List of islands of Italy, nearly 800 islands, notably Sicily and Sardinia. Italy shares land borders with France to the west; Switzerland and Austria to the north; Slovenia to the east; and the two enclaves of Vatican City and San Marino. It is the List of European countries by area, tenth-largest country in Europe by area, covering , and the third-most populous member state of the European Union, with nearly 59 million inhabitants. Italy's capital and List of cities in Italy, largest city is Rome; other major cities include Milan, Naples, Turin, Palermo, Bologna, Florence, Genoa, and Venice. The history of Italy goes back to numerous List of ancient peoples of Italy, Italic peoples—notably including the ancient Romans, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Germany
Germany, officially the Federal Republic of Germany, is a country in Central Europe. It lies between the Baltic Sea and the North Sea to the north and the Alps to the south. Its sixteen States of Germany, constituent states have a total population of over 84 million in an area of , making it the most populous member state of the European Union. It borders Denmark to the north, Poland and the Czech Republic to the east, Austria and Switzerland to the south, and France, Luxembourg, Belgium, and the Netherlands to the west. The Capital of Germany, nation's capital and List of cities in Germany by population, most populous city is Berlin and its main financial centre is Frankfurt; the largest urban area is the Ruhr. Settlement in the territory of modern Germany began in the Lower Paleolithic, with various tribes inhabiting it from the Neolithic onward, chiefly the Celts. Various Germanic peoples, Germanic tribes have inhabited the northern parts of modern Germany since classical ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chiesi Farmaceutici
Chiesi Farmaceutici S.p.A. is an Italian family controlled global pharmaceutical company based in Parma, Emilia-Romagna. Chiesi has 31 affiliates in the world, nearly 6,500 total employees and provides medicines to patients in 100 nations. Chiesi currently has revenues of 3 billion euros. According to 2020 official data from the European Patent Office (EPO), Chiesi Group, with 42 patents filed, is confirmed as the first Italian Pharmaceutical Company and third among Italian Companies across all sectors for filing the highest number of patents. In June 2019, Chiesi became a certified B Corporation, thus choosing to adopt a new legal status under US and Italian law. This ensures Chiesi legal protection to pursue a business model placing social and environmental values on equal footing with profits. Following the acquisition, in 1999, of Huddersfield based Trinity Pharmaceuticals, Chiesi has also had a significant presence in Manchester, United Kingdom. History Established in ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
