Alpha synuclein - A normal protein found in the brain of unknown function, but can aggregate to form insoluble fibrils in pathological conditions characterized by Lewy bodies, such as in Parkinson's disease.

AMD - Age-related Macular Degeneration. A condition that causes severe deterioration of vision and may ultimately cause blindness.

Antisense – A nucleotide sequence that is complementary to a sequence of messenger RNA. When antisense DNA or RNA is added to a cell, it binds to a specific messenger RNA molecule and inactivates it by RNAse H-mediated degradation in the nucleus.

Antisense oligonucleotide – Short single strand of modified nucleotides used for antisense

Antisense therapeutic – Antisense oligonucleotide used therapeutically to downregulate a gene target associated with disease

Base pair - A pair of nucleotide bases which connect either DNA or RNA into a double strand.

Cystic fibrosis - A genetic disease caused by a defective protein known as the cystic fibrosis transmembrane protein (CFTR). Normal CFTR is responsible for the transport of salts and water from inside of cells to extracellular compartments. Mutations in the CFTR gene can result in misfolded CFTR protein that prevents the transport of salts and water. In the lung epithelium, this results in abnormally thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections.

DNA - Deoxynucleic Acid. The chemical information molecule used by most biological systems, which is a string of adenosine (A), cytosine (C), guanine (G), and thymine (T) nucleotides.

dsRNA - Double-stranded RNA. RNA with two strands instead of the typical one.

Enzyme - A protein that regulates a biochemical reaction.

Gene - The basic unit of genetic information, which is coded in the characteristic string of nucleotide bases (coded as A, C, T and G), in a specific sequence that provides the information for making a specific protein.

Genome - The collection of all the genes in the organism.

mRNA - Messenger RNA. Single strands of RNA that transcribe the information contained in DNA to the protein-making machinery of the cell in order to produce specific proteins.

miRNA - microRNA. Naturally expressed small RNAs that interact with components shared by the RNA-induced silencing complex (RISC). It is increasingly believed that microRNAs play a central role in the regulation of gene expression in mammalian cells, and abnormalities in their function may play a role in human disease.

Monoclonal antibody - A single type of antibody that is directed against a specific epitope and is produced by a single clone of B cells or a single hybridoma cell line, which is formed by the fusion of a lymphocyte cell with a myeloma cell.

Nogo - Protein involved in the inhibition of nerve cell regeneration

Parkinson's disease - A neurodegenerative disease of the substantia nigra (an area in the basal ganglia) associated with the destruction of brain cells that produce dopamine and characterized by muscular tremor, slowing of movement, partial facial paralysis, peculiarity of gait and posture, and weakness.

Protein - A primary component of cells, containing linked amino acids whose order is specified by a gene. Proteins build up most of the structures in cells and act as "little machines" that work together inside of cells to accomplish most cellular functions.

Receptor - A protein, usually on the cellular surface, that interacts with binding substances, including drugs, to produce a biological response.

RISC - RNA-induced silencing complex (RISC), forming a naturally stable protein-siRNA complex that can recognize and destroy target mRNAs.

RNA - Ribo-nucleic acid. A naturally-occurring molecule key to essential cellular function which, like DNA, is made up of a string of bases.

RNAi - RNA interference. A naturally occurring biological process of gene silencing

RSV - Respiratory Syncytial Virus. A major cause of respiratory illness particularly in infants and young children as well as other immune deficient populations (e.g. elderly, bone marrow transplant); specifically, infants born prematurely and children with lung or congenital heart disease are at the greatest risk.

shRNA - Short hairpin RNA. A short sequence of RNA that forms a hairpin structure and can be used to silence gene expression at the transcriptional level

siRNA - Small interfering RNA. "Short pieces" of dsRNA, approximately 21-25 base pairs long, which are central to RNAi.

Small molecule - A "small" chemical, often in the form of a drug, which can be absorbed through the gut. Contrasted with "large" protein therapeutics which must be administered by injection to bypass the gut.

Spinal cord injury - A disturbance of the spinal cord that results in loss of sensation and mobility

VEGF - Vascular Endothelial Growth Factor. A substance made by cells that stimulates new blood vessel formation.