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DB-OTO
DB-OTO is an experimental gene therapy for otoferlin-related hearing loss developed by Regeneron Pharmaceuticals. It is delivered via 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 .... References {{reflist Experimental gene therapies ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Otoferlin
Otoferlin is a protein that in humans is encoded by the ''OTOF'' gene. It is involved in vesicle membrane fusion, and mutations in the OTOF gene are associated with a genetic form of deafness. Function There are two forms of otoferlin protein. The short form of the protein has three C2 domains and a single carboxy-terminal transmembrane domain found also in the ''C. elegans'' spermatogenesis factor FER-1 and human dysferlin. The long form has six C2 domains. Dysferlin and myoferlin are proteins found in humans that are homologous to otoferlin. Both dysferlin and myoferlin have seven C2 domains. A C2 domain is a protein structural domain involved in targeting proteins to cell membranes. C2A in otoferlin's longer form, with six C2 domains, is structurally similar to dysferlin C2A. However, loop 1 in the calcium (Ca2+) binding site of otoferlin C2A is significantly shorter than the homologous loop in dysferlin and myoferlin C2A domains. Therefore, it is unable to bind to cal ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Regeneron Pharmaceuticals
Regeneron Pharmaceuticals, Inc. is an American biotechnology company headquartered in Westchester County, New York. The company was founded in 1988. Originally focused on neurotrophic factors and their regenerative capabilities, giving rise to its present name; the company has since expanded operations into the study of both cytokine and tyrosine kinase receptors, which gave rise to their first product, which is a VEGF-trap. Company history The company was founded by CEO Leonard Schleifer and scientist George Yancopoulos in 1988. Regeneron has developed aflibercept, a VEGF inhibitor, and rilonacept, an interleukin-1 blocker. VEGF is a protein that normally stimulates the growth of blood vessels, and interleukin-1 is a protein that is normally involved in inflammation. On March 26, 2012, Bloomberg announced that Sanofi and Regeneron were in development of a new drug that would help reduce cholesterol up to 72% more than its competitors. The new drug would target the PCS ... [...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] |
The Times
''The Times'' is a British Newspaper#Daily, daily Newspaper#National, national newspaper based in London. It began in 1785 under the title ''The Daily Universal Register'', adopting its modern name on 1 January 1788. ''The Times'' and its sister paper ''The Sunday Times'' (founded in 1821), are published by Times Media, since 1981 a subsidiary of News UK, in turn wholly owned by News Corp. ''The Times'' and ''The Sunday Times'' were founded independently and have had common ownership only since 1966. It is considered a newspaper of record in the UK. ''The Times'' was the first newspaper to bear that name, inspiring numerous other papers around the world. In countries where these other titles are popular, the newspaper is often referred to as or , although the newspaper is of national scope and distribution. ''The Times'' had an average daily circulation of 365,880 in March 2020; in the same period, ''The Sunday Times'' had an average weekly circulation of 647,622. The two ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
