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Amino acids
Organic acids that are the building blocks of proteins.
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Anti-angiogenesis
Preventing the formation of blood vessels.
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Antisense
The complementary quality certain RNA or DNA strands possess that enable them to pair with specific strands
of messenger RNA. This pairing prevents the messenger RNA from translating into a protein, thereby preventing
gene functioning. Antisense drug therapy employs this process to prevent malignant cells from multiplying.
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CFTR
Cystic fibrosis transmembrane regulator (CFTR) is a protein. This protein is important in maintaining normal salt and water balance in the lungs, gastrointestinal tract, skin, and other organs. Mutations in the gene for CFTR can result in cystic fibrosis.
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Clinical trials
Clinical trials are medical experiments done in humans to test a new drug or device to understand how well a new drug works and whether it is safe. Most clinical research that involves testing of a new drug progresses in an orderly series of steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects patients. Clinical trials are usually classified into 3 major phases:
- Phase 1 trials: The primary purpose of these initial studies is to determine an appropriate and safe dosing regimen for use in future studies in patients. These studies will also assess the safety and pharmacokinetics of a new drug. If the drug is given orally, they may determine whether taking food with the drug alters how much drug is absorbed. These studies are the first studies in people. They are usually performed at a single specialized center and include only small number of participants (often healthy volunteers). Such studies may or may not include a placebo group or another standard type of treatment as a control.
- Phase 2 trials: The primary goal of these studies is to understand whether a new drug is active against its intended molecular target. Building on the Phase 1 data, these studies evaluate that activity while obtaining additional short-term safety and pharmacokinetic information. These studies are usually performed at several clinics or hospitals working in collaboration. A few dozen to several hundred patients with a specific disease are typically included, most often in a single country. Such studies may or may not include a placebo group or another standard type of treatment as a control.
- Phase 3 trials: The primary objective of these studies is to prove that a drug, when given at a dose shown to be active in Phase 2, offers clinical benefit to patients by prolonging life, reducing symptoms, or preventing disease. These studies usually test the new drug in comparison to a placebo or to a currently available therapy. Participants will most commonly be assigned to the standard group or the new group at random. For rare diseases, small Phase 3 trials may be possible. For common diseases, Phase 3 trials will often enroll hundreds to thousands of patients with a specific disease and may be conducted at many doctors' offices, clinics, and medical centers internationally.
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Cystic fibrosis
A hereditary disease that causes disorder of the exocrine glands. Effects of the disease include a
lack of pancreatic enzymes resulting in flawed digestion, difficulty breathing due to mucus accumulation
in airways, and loss of salt in sweat. Cystic fibrosis is prevalent in Caucasians and usually emerges in early childhood.
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Cytoplasm
The matter inside a cell that surrounds the nucleus and in which the organelles of the cell are suspended.
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Deoxyribonucleic acid (DNA)
DNA is a nucleic acid that constitutes the genetic material of all living organisms. To be useful to the cell, the genetic code in DNA is copied to messenger RNA by the process of transcription.
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Duchenne Muscular Dystrophy (DMD)
A severe progressive form of muscular dystrophy limited to males. DMD is an X-linked recessive
trait and marked by the absence of the protein dystrophin. DMD affects the leg muscles before it
affects the arm muscles, and affects proximal muscles in the limbs before the distal muscles. It
appears in early childhood and is usually fatal by age 20.
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Dystrophin
Dystrophin is a long structural protein important for maintaining the strength of muscle fibers. Mutations in the gene for dystrophin can result in Duchenne or Becker muscular dystrophy.
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GEMS Technology
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Genome
The complete genetic makeup of an organism. Also, a haploid set of chromosomes
with all of the genes they contain.
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Hepatitis C
A disease of the liver caused by a single-stranded RNA virus of the family Flaviviridae (species Hepatitis C virus of the genus Hepacivirus). Hepatitis C tends to persist in the
blood serum, accounts for most cases of non-A and non-B hepatitis, and is most often
transmitted by exposure to infected blood or blood products, including injection of an
illicit drug and blood transfusion.
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Internal ribosome entry site (IRES):
An access point through which ribosomes can initiate translation of messenger RNA (mRNA) without the necessity of commencing at the 5’ capped end. Such cap-independent translation allows the mRNA of several pathogens, such as that from Hepatitis C Virus (HCV), to compete effectively with cellular mRNAs.
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In vivo
In vivo describes a process or a scientific experiment occurring within a living animal or human body.
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In vitro
In vitro describes a process or scientific experiment occurring within an artificial environment, such as a Petri dish or test tube.
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Messenger Ribonucleic acid (RNA):
Messenger RNA, also known as mRNA,
provides a copy of the genetic material contained in DNA. Components of the cellular machinery known as ribosomes translate the genetic code contained in the mRNA to build long chains of molecules known as proteins. Proteins are molecules that serve as the building blocks and regulators of all life.
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Nasal transepithelial potential difference (TEPD)
TEPD is a standardized procedure in which a small plastic catheter is used to assess electrical differences across the cell membrane of the skin in each nostril. This test assesses how much of the CFTR chloride channel is present. In patients with cystic fibrosis, the TEPD chloride conductance is usually abnormal because there is very little functional CFTR present.
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Nonsense mutation
A nonsense mutation is an alteration in DNA which, when copied to mRNA, inserts a premature stop codon into the mRNA. This premature stop codon signals the ribosomes to stop production of a particular protein too early in the translation process. Such a mutation in the gene that codes a crucial protein can cause disease because the resulting protein can be too short to serve its necessary biological function.
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Nucleotide:
Any compound that consists of a ribose or deoxyribose sugar attached to a purine or
pyrimidine base and to a phosphate group. Nucleotides are the building blocks of RNA and DNA
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Pharmacokinetics:
Pharmacokinetics describe the movement of a drug through the body over a period of time. Most commonly pharmacokinetics are used to define the amount of drug circulating in the blood stream as a measure of how the drug is taken up by the body and delivered by the blood stream to the site of action.
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Polypeptide:
A molecular chain of a large number of amino acids that are connected by peptide (-NH-CO-) links.
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Post-transcriptional control processes
The sequence of events in a cell that regulate the rate and timing of all protein production.
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Protein
Any naturally occurring complex substance, such as an enzyme or antibody, that consists of amino acid
residues joined by peptide bonds, and containing the elements carbon, hydrogen, nitrogen, oxygen, and
often sulfur. They also occasionally contain other elements, such as phosphorus or iron. Proteins are
essential constituents of all living cells that are synthesized from raw materials by plants, but
assimilated as separate amino acids by animals.
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Ribose
The monosaccharide C5H10O5 that forms the side chains of ribonucleic acid (RNA) molecules.
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Ribosome
A ribosome is a part of the cellular machinery responsible for translating mRNA to make proteins.
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RNA interference (RNAi):
A post-transcriptional genetic mechanism that involves the silencing of gene expression. RNAi is triggered
when double-stranded RNA cleaves into small fragments initiates the degradation of a complementary messenger RNA.
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Small-molecule drugs
Drugs that can be taken orally, unlike proteins, which must be administered by injection or topically. The name is due to these drugs' low molecular weight.
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Transcription
The construction of a messenger RNA (mRNA) molecule using a DNA molecule as a template. During this process, the genetic information of the DNA is copied onto the mRNA.
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Translation
Translation is the process by which proteins are synthesized by ribosomes using the mRNA as a template.
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Untranslated regions (UTRs)
Regions of mature RNA that are not involved in protein synthesis. UTRs contain information for regulation of translation and mRNA stability.
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Vascular endothelial growth factor (VEGF):
A protein that plays a significant part in promoting the development of new blood vessels.
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