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Icahn Institute For Genomics And Multiscale Biology
The Icahn Genomics Institute is a biomedical and genomics research institute located in New York, NY. It is housed within the Icahn School of Medicine at Mount Sinai. Its aim is to establish a new generation of medicines that can better treat diseases afflicting the world, including cancer, heart disease and infectious pathogens. To do this, the institute’s doctors and scientists are developing and employing new types of treatments that utilize DNA and RNA based therapies, such as CRISPR, siRNA, RNA vaccines, and CAR T cells, and searching for novel drug targets through the use of functional genomics and data science. The institute is led by Brian Brown, a leading expert in gene therapy, genetic engineering, and molecular immunology. Goals The institute’s primary goal is to improve patient care through the use of gene, cell and nucleotide therapies. To achieve this goal, the Institute is formed of a cross-disciplinary mix of clinicians and scientists that include physicians ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Translational Research
Translational research (also called translation research, translational science, or, when the context is clear, simply translation) is research aimed at translating (converting) results in basic research into results that directly benefit humans. The term is used in science and technology, especially in biology and medical science. As such, translational research forms a subset of applied research. The term has been used most commonly in life-sciences and biotechnology but applies across the spectrum of science and humanities. In the context of biomedicine, translational research is also known as bench to bedside. In the field of education, it is defined as research which translates concepts to classroom practice. Critics of translational medical research (to the exclusion of more basic research) point to examples of important drugs that arose from fortuitous discoveries in the course of basic research such as penicillin and benzodiazepines. Other problems have stemmed from th ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
PLOS Biology
''PLOS Biology'' is a monthly peer-reviewed scientific journal covering all aspects of biology. Publication began on October 13, 2003. It is the first journal published by the Public Library of Science. The editor-in-chief is Nonia Pariente. In addition to research articles, the journal publishes magazine content aimed to be accessible to a broad audience. Article types in this section are essays, "unsolved mysteries", editorials, and synopses. Abstracting and indexing The journal is abstracted and indexed in: * Biological Abstracts * BIOSIS Previews *'' Current Contents''/Agriculture, Biology & Environmental Sciences *''Current Contents''/Life Sciences *Chemical Abstracts Service * Embase *''Index Medicus''/MEDLINE/PubMed *Science Citation Index *Scopus *''The Zoological Record'' According to ''Journal Citation Reports'', the journal had a 2019 impact factor of 7.076. The journal does not list this impact factor on its website. Instead, the journal promotes the use of article ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS is the most common type of motor neuron diseases. Early symptoms of ALS include stiff muscles, muscle twitches, and gradual increasing weakness and muscle wasting. ''Limb-onset ALS'' begins with weakness in the arms or legs, while ''bulbar-onset ALS'' begins with difficulty speaking or swallowing. Half of the people with ALS develop at least mild difficulties with thinking and behavior, and about 15% develop frontotemporal dementia. Most people experience pain. The affected muscles are responsible for chewing food, speaking, and walking. Motor neuron loss continues until the ability to eat, speak, move, and finally the ability to breathe is lost. ALS eventually causes paralysis and early death, usually from respiratory failure. Most cases of ALS (a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bispecific Antibodies
A bispecific monoclonal antibody (BsMAb, BsAb) is an artificial protein that can simultaneously bind to two different types of antigen or two different epitopes on the same antigen. Naturally occurring antibodies typically only target one antigen. BsAbs can be manufactured in several structural formats. BsAbs can be designed to recruit and activate immune cells, to interfere with receptor signaling and inactivate signaling ligands, and to force association of protein complexes. BsAbs have been explored for cancer immunotherapy, drug delivery, and Alzeimer's disease. Development history The original concept of BsAbs was proposed by Nisonoff and his collaborators in the 1960s, including the first idea of antibody architecture and other findings. In 1975, the problem of producing pure antibodies was solved by the creation of hybridoma technology, and the new era of monoclonal antibodies (MoAbs) came. In 1983, Milstein and Cuello created hybrid-hybridoma (quadroma) technology. In 1988 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chimeric Antigen Receptor
In biology, chimeric antigen receptors (CARs)—also known as chimeric immunoreceptors, chimeric T cell receptors or artificial T cell receptors—are receptor proteins that have been engineered to give T cells the new ability to target a specific antigen. The receptors are chimeric in that they combine both antigen-binding and T cell activating functions into a single receptor. CAR T cell therapy uses T cells engineered with CARs to treat cancer. The premise of CAR T immunotherapy is to modify T cells to recognize cancer cells in order to more effectively target and destroy them. Scientists harvest T cells from people, genetically alter them, then infuse the resulting CAR T cells into patients to attack their tumors. CAR T cells can be derived either from T cells in a patient's own blood (autologously) or from the T cells of another, healthy, donor ( allogeneically). Once isolated from a person, these T cells are genetically engineered to express a specific CAR, which programs ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
T Cell
A T cell is a type of lymphocyte. T cells are one of the important white blood cells of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell receptor (TCR) on their cell surface. T cells are born from hematopoietic stem cells, found in the bone marrow. Developing T cells then migrate to the thymus gland to develop (or mature). T cells derive their name from the thymus. After migration to the thymus, the precursor cells mature into several distinct types of T cells. T cell differentiation also continues after they have left the thymus. Groups of specific, differentiated T cell subtypes have a variety of important functions in controlling and shaping the immune response. One of these functions is immune-mediated cell death, and it is carried out by two major subtypes: CD8+ "killer" and CD4+ "helper" T cells. (These are named for the presence of the cell surface proteins ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Multiple Myeloma
Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, kidney dysfunction, and infections may occur. Complications may include amyloidosis. The cause of multiple myeloma is unknown. Risk factors include obesity, radiation exposure, family history, and certain chemicals. There is an increased risk of multiple myeloma in certain occupations. This is due to the occupational exposure to aromatic hydrocarbon solvents having a role in causation of multiple myeloma. Multiple myeloma may develop from monoclonal gammopathy of undetermined significance that progresses to smoldering myeloma. The abnormal plasma cells produce abnormal antibodies, which can cause kidney problems and overly thick blood. The plasma cells can also form a mass in the bone marrow or soft tissue. When one tumor is p ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Blood (journal)
''Blood'' is a peer-reviewed medical journal published by the American Society of Hematology. It was established by William Dameshek in 1946. The journal changed from semimonthly (24 times annually) to weekly publication at the start of 2009. It covers clinical and basic research in all areas of hematology, including disorders of leukocytes, both benign and malignant, erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Abstracting and indexing ''Blood'' is indexed and abstracted by: According to the ''Journal Citation Reports'', the journal has a 2021 impact factor The impact factor (IF) or journal impact factor (JIF) of an academic journal is a scientometric index calculated by Clarivate that reflects the yearly mean number of citations of articles published in the last two years in a given journal, as ... of 25.476, and has a Journal Impact Factor rank of 1/76 in the Hematology category. References External ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Tumor Growth Factor (TGF) Beta
Transforming growth factor beta (TGF-β) is a multifunctional cytokine belonging to the transforming growth factor superfamily that includes three different mammalian isoforms (TGF-β 1 to 3, HGNC symbols TGFB1, TGFB2, TGFB3) and many other signaling proteins. TGFB proteins are produced by all white blood cell lineages. Activated TGF-β complexes with other factors to form a serine/threonine kinase complex that binds to TGF-β receptors. TGF-β receptors are composed of both type 1 and type 2 receptor subunits. After the binding of TGF-β, the type 2 receptor kinase phosphorylates and activates the type 1 receptor kinase that activates a signaling cascade. This leads to the activation of different downstream substrates and regulatory proteins, inducing transcription of different target genes that function in differentiation, chemotaxis, proliferation, and activation of many immune cells. TGF-β is secreted by many cell types, including macrophages, in a latent form in which it ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Givosiran
Givosiran, sold under the brand name Givlaari, is a medication used for the treatment of adults with acute hepatic porphyria. Givosiran is a small interfering RNA (siRNA) directed towards delta-aminolevulinate synthase 1 (ALAS1), an important enzyme in the production of heme. The most common side effects include nausea and injection site reactions. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Givosiran is indicated for the treatment of adults with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood). History In November 2019, givosiran was approved in the United States for the treatment of adults with acute hepatic porphyria (AHP). Efficacy was evaluated in ENVISION (NCT03338816), a randomized, double‑blind, placebo‑controlled, multinational trial enrolling 94 participants wi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hepatic Porphyria
Hepatic porphyrias is a form of porphyria in which toxic porphyrin molecules build up in the liver. Hepatic porphyrias can result from a number of different enzyme deficiencies. Examples include (in order of synthesis pathway): * Acute intermittent porphyria * Porphyria cutanea tarda and Hepatoerythropoietic porphyria * Hereditary coproporphyria * Variegate porphyria See also * Erythropoietic porphyria * Givosiran Givosiran, sold under the brand name Givlaari, is a medication used for the treatment of adults with acute hepatic porphyria. Givosiran is a small interfering RNA (siRNA) directed towards delta-aminolevulinate synthase 1 (ALAS1), an important ... References External links * * www.drugs-porphyria.com * www.porphyria-europe.com Porphyrias {{endocrine-disease-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
