In the human body, proteins are essential to maintain and regulate all biological functions. Many diseases are caused by the defective production of proteins. Proteins can be under- or overproduced, or defective, disease causing proteins can be formed.
The information for all proteins is stored in the DNA, with a copy in every single cell. If a certain protein has to be produced, a copy of the part of the DNA with the construction plan of this protein is made, called mRNA. This mRNA is then transported to cellular compartments called ribosomes where it is “translated” into proteins. A failure in this process, e.g. a mutation in the construction plan, can cause the formation of a defective disease-causing protein.
Antisense molecules allow intervening at a very early stage in the development of a disease: they prevent the formation of the disease-causing proteins. Antisense molecules which fit exactly to a certain segment of the mRNA bind to it and block he translation into the disease-causing proteins.