For your convenience, below is a list of terms used on this site and elsewhere in relation to RNA interference. Please contact us if you would like to suggest any addition to this list.
| AAV | Adeno-associated virus - a small virus commonly used as a vector in gene therapy |
| adenovirus | A medium sized dsRNA virus first isolated from human adenoids |
| anti-sense | RNA or DNA sequence synthesized to be complementary to the normal or 'sense' mRNA sequence |
| cytoplasm | The part of the cell containing the organelles which excludes the nucleus |
| ddRNAi | DNA-directed RNA interference – RNAi expressed from a DNA construct. Benitec Biopharma's patented technology. shRNA is an example of ddRNAi. |
| ddRNAi therapeutics | Human therapeutics based on ddRNAi, Benitec Biopharma's patented process |
| Dicer | An enzyme in the cell's cytoplasm that cuts dsRNA into double stranded small interfering (sui)RNA molecules 20-25 nucleotides long. |
| DNA | Deoxyribonucleic acid – the chemical that comprises the genetic blueprint for all organisms (except RNA viruses) |
| DNA cassette | A fragment of DNA carrying and capable of expressing one or more DNA sequences coding for RNA sequences of interest |
| DNA construct | A synthesized segment of DNA to be transplanted (or transfected) into the DNA of another cell |
| dsRNA | double stranded RNA – two sequences of RNA that are complementary to each other. dsRNA usually only occurs in cells as part of RNA interference |
| genes | Those stretches of DNA that code for proteins or RNA sequences - the organism's unit of heredity |
| gene cassette | A manipulable fragment of DNA carrying and capable of expressing, one or more genes of interest |
| gene silencing | The knocking down of a gene so that its usual product e.g. a protein, is not formed |
| gene therapy | Use of introduced DNA to replace a mutated gene, to correct a mutation or code for a therapeutic protein |
| genetic disease | Illness or condition caused by abnormalities in genes or chromosomes; may be both heritable and non-heritable |
| guide strand | The strand from unwound siRNA which binds to RISC in RNAi. The other strand (the passenger) is degraded.. |
| immune activation | Activation of the immune (defence) response by the cell |
| lentivirus | A virus often used as a gene therapy vehicle to introduce DNA into target cells |
| mRNA | messenger RNA - a molecule of RNA which is transcribed from DNA and is translated into a protein |
| NSCLC | non-small cell lung cancer - a type of epithelial lung cancer which is relatively insensitive to chemotherapy, compared to small cell carcinoma |
| nucleotides | The structural units of DNA and RNA, made up of a nitrogenous base, a 5 carbon sugar and a phosphate group |
| nucleus | The part of the cell that is not the cytoplasm in eukaryotes (prokaryotes only have cytoplasm and no nucleus) |
| off target effects | The switching off of unintended gene functions as a result of gene silencing |
| opiod infusion | Infusion of opioids (e.g. morphine) for pain management usually in terminal patients |
| OPMD | oculopharyngeal muscular dystrophy - a rare, slow progressing, degenerative disease muscle disorder; characterized by progressive eyelid drooping and swallowing difficulties. |
| passenger strand | The strand from unwound siRNA in RNAi which is degraded; the other (the guide strand) binds to the RISC |
| polyethylenimine | PEI - a polymeric chemical used to deliver DNA into target cells |
| RNAi | RNA interference - a natural regulatory process in the cell to turn off unwanted genes |
| RNA-Induced Silencing Complex | RISC - an enzyme complex in the RNAi pathway which binds siRNA and then separates it into guide and passenger strands |
| shRNA | short hairpin RNA formed by two short complementary sequences of RNA separated by a non-complementary sequence (the hairpin), expressed from a ddRNAi DNA construct |
| siRNA | small interfering RNA - an intermediate in the RNAi pathway; occurs transiently in expressed RNAi from shRNA molecules after Dicer processing and may also be synthesized in a laboratory and introduced to the cell externally |
| ssRNA | single stranded RNA that occurs naturally in RNA viruses or is an intermediate in cellular pathways |
| sshRNA | Synthetic short hairpin RNA molecule - a shRNA which is synthesized in a laboratory and introduced externally into the target cell |
| systemic injection | Injection into the blood stream for delivery to the whole body or a specific organ or tissue therein |
| transfection | The process of introducing nucleic acids into cells |
| vectors | Molecules (viral and non) used to introduce nucleic acids into cells |
| zinc finger nucleases | ZFNs - artificial restriction enzymes used as reagents to target specific DNA sequences in the cell nucleus. Can only be introduced into cells ex vivo. |
