Laboratory of Molecular Design
Antisense Therapy


anti       If a particular gene has a role in disease, and the genetic code of that gene is known, one could use this knowledge to stop that gene specifically. Genes are made of double-helical DNA. When a gene is turned on, the genetic code in that segment of DNA is copied out as a single strand of RNA, called messenger RNA. The messenger RNA is called a "sense" sequence, because it can be translated into a string of amino acids to form a protein. The opposite strand in a DNA double helix (A opposite T, T opposite A, C opposite G, G opposite C) is called the "antisense" strand. We use the antisense coding sequence of a disease gene to make short antisense DNAs in our laboratory. These antisense DNA drugs work by binding to messenger RNAs from disease genes, so that the genetic code in the RNA cannot be read, stopping the production of the disease-causing protein.

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