Niemann–Pick type C (NPC) disease is a rare progressive genetic disorder, characterized by the inability of the body to transport lipids and cholesterol inside the cells.
NF-κB is identified as a family of highly conserved transcription factors, which are responsible for many cellular functions such as regulation of inflammatory responses, apoptosis, cellular growth, and others.
IL-6 is transcribed by IL-6 gene that encodes a cytokine, which functions in inflammation and maturation of B cells. The IL-6 protein can induce fever in patients suffering from autoimmune diseases, thereby acting as endogenous pyrogens.
BPH is a non-cancerous medical condition, which is characterized by enlargement of prostate gland. The first phase of BPH starts in puberty, wherein the size of prostate gland becomes double.
Wnt or β-Catenin signaling pathway plays a key role in the development of pluripotent cells and embryonic development. The pathway regulates cell-to-cell interaction, activates blastocyst, accelerates trophoblast development, accelerate chorion-allantois fusion and implantation.
Galectins belong to the family of animal lectins and perform function by interacting with cytoplasmic and nuclear proteins, cell-surface and extracellular matrix glycoproteins and glycolipids to modulate signalling pathways.
Epidermal growth factor receptors (EGFR) are a group of receptor tyrosine kinase (RTK) that plays a major role in cell proliferation, survival and differentiation. In most of the cancer, EGFR are amplified and overexpressed, due to dysregulation, resulting in cancer development.
Endothelin antagonists are drug candidates that acts against endothelin receptors and generate pharmacological actions such as blocking the vasoconstriction and mediate vasodilatation. Endothelin-1 is a peptide which is comprised of 21 amino acids and formed by vascular endothelium.
Mitogen-activated protein kinase (MAPK) are ubiquitous regulators of many cellular functions including cell proliferation, cellular growth, inflammatory responses to stress signals and cell differentiation.
MDM2 protein are powerful oncogene which is overexpressed in various cancers, including breast cancer and sarcoma. There are many small molecule drug candidates that are being developed as MDM2 protein inhibitors as monotherapy or combination therapy for the treatment of various cancers.
Interleukin receptors are a group of cytokine receptors that are expressed by leukocytes. Interleukin receptors play an important role in the functioning of the immune system.
Hepatocyte growth factor (HGF) is secreted by human pancreatic stellate cells and present predominantly on cancer cells. Thus, HGF inhibitors has emerged as an effective therapy in preventing local tumor growth.
Glycogen synthase kinase 3 beta (GSK-3β) is an attractive target for the treatment of psychiatric disorders and neurodegenerative diseases. Many GSK-3β inhibitors have been developed for the treatment of different central nervous system disorders.
Spleen tyrosine kinase (Syk) is a protein that belongs to the family of tyrosine kinases with an ability to initiate the inflammatory responses, by connecting immune cell receptors to intracellular signaling pathways.
RNA interference (RNAi) is a molecule type that silences the gene and limits the transcription of the mutated gene. Gene silencing is a novel mechanism that inactivates the transcripts of mutated gene, by activating sequence specific RNA degradation process.
Rho kinase inhibitor, also known as ROCK inhibitor, inhibits the rho kinases, a family of small GTP-binding protein. Rho kinases are found to regulate cell motility, proliferation, shape, gene expression, apoptosis, and are also involved in the signalling pathway.
p53 is the tumor suppressor transcription factor that activates to various stimulus, including uncontrolled cell proliferation, oncogene over-expression, and DNA damage. p53 antigen modulators helps in preventing cancer development through regulation of cell cycle and apoptosis.
Nav1.7 voltage-gated sodium channel inhibitors are the drug candidates that target the sodium channels Nav1.7 and have been significant in pain management. The sodium channels Nav1.7 receptors are generally found in two types of neurons: nociceptive pain neurons, such as trigeminal neurons and dorsal root ganglion; and sympathetic ganglion neurons which form a part of the autonomic (involuntary) nervous system. These receptors play an important role in generation and conduction of action potential.
Vitamins are nutrients that are required for the growth of the body. Based on its solubility, vitamins are categorized into two types: fat soluble vitamins, and water-soluble vitamins. Vitamins have a variety of function in the body, such as vitamin A improves eye sight, vitamin D is essential for bone growth and has hormone like function that regulates mineral metabolism.
A vaccine is a biological preparation that modulates the immunity in the body, in response to a particular disease. It is composed of a disease-causing microorganism, which is in killed form, and toxins produced by the microorganism.
Recombinant biologic is an entity that includes wide range of therapeutics, such as blood and blood components, vaccines, tissues, gene therapy, and recombinant therapeutic proteins.
Protein is a large biomolecule, composed of one or more long chains of amino acids, known as polypeptide. Proteins play an important role in metabolic reactions, transporting molecules, and DNA replication.
Probiotics are live microorganisms that produces beneficial effects in human gut. Probiotics can be taken as a dietary supplement. They prevent diarrhoea and help to reduce the symptoms of irritable bowel syndrome.
Peptides are structurally similar to protein, comprising of peptide bonds, but smaller than proteins. A peptide bond is formed when two amino acids are combined through amide formation (combination of carboxyl group of one amino acid and amino group of the other amino acid).
Nano therapy is a technique derived from nanotechnology, with improved drug solubility and half-life, and enhanced targeted delivery. The size of a nanoparticle ranges from 5 to 500 nm in one dimension.
Monoclonal antibodies are the prominent therapeutic agents in biomedical research, as they are implemented in the diagnosis and treatment of various diseases areas, including cancer, infectious diseases, immunology, and cardiovascular disorders.
Lipids are the large and miscellaneous group of organic compounds that are characterized by its solubility in non-polar solvents. There are various types of lipids, such as fatty acids, fats, oils, waxes, soaps and detergents, eicosanoids, terpenes, steroids, and phospholipids.
Gene therapy is a technique that involves treatment or prevention of a disease by using genes. Several methodologies are being used in the gene therapy that involve replacing the mutated gene, inactivating or silencing the mutated gene, and introducing a new gene into the body.
Cell therapy is a type of therapy in which living cells are administered into a patient’s body for the treatment of a disease. Stem and progenitor cells have shown potential in cell therapy for the treatment of several pathological conditions.
Chimeric antigen receptor T (CAR T) therapy is designed with a single signaling domain. CAR T therapy is comprised of engineered T-cells, that produce chimeric antigen receptor on their cell membrane.
Cannabinoid is a type of alkaloid that is obtained from the cannabis sativaplants. Cannabis, also known as marijuana, produce resin containing compounds known as cannabinoids. Cannabinoids are being used for medical purposes from the ancient times.
Biosimilar are biological products that show similar biological action as the U.S. Food and Drug Administration (USFDA) approved biologics. According to the U.S. Federal Code of Regulation, the biologic is a virus, toxin, antitoxin, or therapeutic serum valid for the treatment of a disease.
Cytotoxic T-lymphocyte antigen-4 (CTLA-4) plays important role in the T-cell regulation at an early stage of naive T-cell activation, primarily in the lymph nodes. CTLA-4 has emerged as an attractive cancer immunotherapy as a part of “immune checkpoint blockade”.
Cyclophilin befits in the group of protein that shows peptidyl-prolyl cis-trans isomerase activity, found in all types of cells. In humans, 16 cyclophilins have been identified till date. Cyclophilin A, a member of cyclophilin group, mediate the action of immunosuppressive drugs.
Cell cycle inhibitors include cyclin inhibitors and cyclin-dependent kinases (CDKs), which plays major role in developing new class of anti-cancer therapies. Also, cell cycle inhibitors in combination with chemotherapy, overcome drug resistance and improve cytotoxic efficacy. CDKs are rational targets for cancer treatment, that could restore cell-cycle checkpoints and may induce apoptosis.
Cathepsin K is a protease enzyme which is coded by CTSK gene. The function associated with the gene involves bone remodelling and resorption. Cathepsin K is a member of peptidase C1 protein family and expressed mainly in osteoclasts.
Aurora kinase are proteins that function as mitotic regulators and are aberrantly expressed in cancerous cells. Aurora kinase inhibitors target these aberrantly expressed regulators, provides genetic stability and prevents tumorigenesis.
Angiopoietin (ANG) receptor plays a crucial role in regulation of vascular permeability and pathological vascular remodelling during tumour angiogenesis, metastasis and inflammation. Thus, it has emerged as a new clinical approach for the development of therapeutics which treat cancer and ophthalmological diseases.
Adenosine 5-monophosphate activated protein kinase (AMPK) plays an important role in regulation of homeostasis. Dysregulation of AMPK can result in many diseases which includes obesity and type 2 diabetes. Several studies have also suggested that AMPK are also associated with the development of neurological diseases and cancer.
Androgens are responsible for the development of male characteristics by binding to androgen receptors. Androgen receptor plays an important role in the development and progression of prostate cancer. Thus, it has been an attractive target for the clinical interventions and treatment of prostate cancer.
Alpha-synuclein is a protein that is abundantly found in the brain. The minimal amount of alpha-synuclein is also found in the heart and other tissues. The protein is prominently found at the tips of the nerve cells. It helps in the maintaining supply of the synaptic vesicle in presynaptic terminals. In addition, the alpha-synuclein protein also helps in release of dopamine.
Osphena (Ospemifene), an estrogen receptor agonist with tissue selective effects. It is prescribed for painful intercourse (dyspareunia), which is a major symptom of vulvular and vagina muscular wasting (vaginal atrophy), due to menopause.
Prader-Willi syndrome (PWS) is a complicated genetic condition which can affect various parts of the body. During infancy, this condition is identified by weak muscle tone (hypotonia), problems in feeding, poor growth, and delay in development.
Adrenoleukodystrophy (ADL) is defined as a X-linked disorder which is more prominent in males. This disorder affects the adrenal glands. In this disease the fatty covering over the neurons and spinal cord starts deteriorating, thus reducing nerve’s ability to transfer the information. The damage to adrenal glands leads to reduction in the level of adrenocortical hormones.
Hyperkalemia is referred to as increased levels of potassium in blood which results in cardiac arrest and death. Potassium levels above 5.1 mEq/l are considered as the hyperkalemia condition. The main causes of hyperkalemia are potassium sifting out of cells into the blood circulation, adrenal gland diseases, kidney dysfunction, uncontrolled diabetes, breakdown of muscle tissue and red blood cells.
Akt, also known as PKB, is generally activated in several types of cancers such as breast, gastric, lung, ovary, and pancreatic cancer. Akt inhibitor prevent the functioning of activated form of Akt kinases. Akt kinases are also involved in promoting growth factor-mediated cells that mediates cell proliferation and inhibition of apoptosis through the inactivation of pro-apoptotic proteins, such as mouse double minute 2 homolog (MDM2) and Bcl-2 antagonist of cell death.
Achondroplasia, also termed as ACH and achondroplastic dwarfism, is an autosomal dominant genetic disorder that leads to short-stature/ limb dwarfism. It is caused due to abnormal cartilage formation. Cartilage is responsible for the development of skeleton and initial bone growth.
Wiskott-Aldrich syndrome is a hereditary immunodeficiency disorder characterized by abnormal functioning of immune system and decreased ability to form blood clots. The abnormality in the platelets leads to easy bruising effects in case of prolonged bleeding.
Vaginitis refers to the inflammation of vagina. It affects women of all ages but is most common during the reproductive years. Vaginitis occurs due to change in the balance of the yeast and bacteria that normally live in the vagina.
Uterine fibroids, also known as leiomyomas or myomas, are non-cancerous growths that develop from the muscle tissue of the uterus. The size, shape, and location of fibroids can vary greatly.
Turner syndrome is a chromosomal condition, which occurs when one of the two X chromosomes normally found in women is missing or incomplete. The disease alters the growth and development in females.
Reperfusion injury is the damage to tissues caused when blood supply returns to the tissue after a period of ischemia or lack of oxygen (anoxia, hypoxia). It influences the patient’s outcome after myocardial infarction, stroke, cardiovascular surgery, and organ transplantation.
Post-operative nausea and vomiting is one of the complications of anaesthesia given to the patients at the time of a surgery. The state of having unpleasant sensation subjected to desire of vomiting is known as nausea whereas, vomiting is the forceful expulsion contents present in gastrointestinal tracts.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a progressive disorder that primarily affects brain and spinal cord (central nervous system). People with MPS III generally do not display features at birth, but they begin to show signs and symptoms of this disorder during early childhood.
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects various parts of the body and occurs almost exclusively in males. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals.
Mucopolysaccharidosis type I (MPS I) is a condition that affects many parts of the body. Earlier, MPS I disorder was divided into three separate syndromes; Hurler syndrome (MPS I-H), Hurler-Scheie syndrome (MPS I-H/S), and Scheie syndrome (MPS I-S), listed from most to least severe. Later on, these were clubbed as one disease, due to overlap between each of these three syndromes.
Ischemia reperfusion injury is a medical condition which involves tissue injury, resulting in the interruption of blood supply, and occurs when the blood supply to an area of tissue is cut off. Ischemia-related tissue injury can occur in various organs including heart and brain. During reperfusion injury, inflammatory responses occur including release of free radicals, vascular leakage, recruitment of inflammatory cells, and proteins that cause tissue destruction.
Infertility is defined as the biological inability to conceive or contribute to conception, after having regular unprotected sex. According to the data published by the Department of Health and Human Services, approximately 10% to 15% of couples in the U.S. are infertile and have not conceived after at least one year of regular, unprotected sex
Hypoxic-ischemic encephalopathy is a serious birth complication that causes obstruction in blood flow in brain during the prenatal, intrapartum or postnatal period. This leads to death of the child or mental disabilities in the first two years of age.
Hypoxia is a condition characterized by inability of the tissues to receive an adequate oxygen supply. This condition is categorized into four types; hypoxic, stagnant, anaemic, fulminating, and histotoxic hypoxia.
Hypoxia-inducible factor (HIF) inhibitor inhibits the HIF mediated hypoxic responses. HIF is a transcription factor that activate when there is a decrease in the availability of oxygen in the cellular environment. Hypoxia promotes the formation of blood vessels and this homeostatic system is required in formation of vascular system in the embryo.
Hyperphosphatemia is a medical condition which involves increased level of serum phosphate concentration in the blood. Phosphate is required for bone and cell formation, genetic coding and energy metabolism. An individual is said to have hyperphosphatemia, if the phosphate concentration increasing beyond > 4.5 mg/dL (> 1.46 mmol/L).
Prostate cancer occurs when cells in prostate glands grow uncontrollably. Prostate is an exocrine gland that makes the fluid part of semen and lie below the bladder in front of the rectum. As per the National Institute of Health (NIH), 10.7% of all new cases of cancer in the U.S. are of prostate cancer.
Hormonal replacement therapy is a type of treatment wherein patients receive a course of hormones in order to get relieve from the menopause related problems. The therapy involves administration of synthetic estrogen and progesterone to overcome decreasing hormone level of women.
Heterozygous familial hypercholesterolemia (HeFH) is an inherited genetic disorder that affects the body’s ability to control cholesterol. It is characterized by very high LDL (low density lipoprotein) cholesterol (above 190 for adults or above 160 for children) and family history of high cholesterol, heart disease or stroke.
Mobilization is the recruitment of hematopoietic stem cells (HSC) into peripheral blood from the bone marrow following chemotherapy treatment. There are several advantages for reinfusion of autologous mobilized peripheral blood stem cells over bone marrow HSC.
Globoid cell leukodystrophy, also known as Krabbe disease, is a genetic disorder which is characterized by decreased production of galactocerebrosidase. The symptoms observed during Krabbe disease are fever, vomiting, loss of head control, irritability and excessive crying, seizures, poor coordination of movement or stiffness, muscle spasms, changes in muscle tone, deterioration of motor function, difficulty walking and muscle weakness.
Female sexual dysfunction (FSD) prevents the females to experience satisfaction from the sexual activity. FSD can be developed during any phase of the sexual response cycle which consists of excitement, plateau, orgasm and resolution phases.
Female contraception prevents unwanted or unintended pregnancy. When the matured egg cell leave the ovary and ovulation begins, the chances of becoming pregnant increases. The main advantage of using contraceptives is reduction in menstrual bleeding and period pain.
Epidermolysis bullosa (EB), is a rare genetic connective tissue skin disorder that causes blisters and allows skin to become fragile. Blisters and areas of skin loss (erosions) occur in response to minor injury or friction, such as rubbing or scratching.
Dental pain, also known as odontogenic pain or tooth pain, arises from the teeth or their supporting structures which includes mucosa, gingivae, maxilla, mandible, or periodontal membrane. The primary location of the reflected pain (orofacial pain) is the spinal core of trigeminal system.