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Gene therapy, artwork
Gene therapy. Diagram showing exon skipping as a means of gene therapy. Genes are sections of DNA (deoxyribonucleic acid) that encode the proteins that determine each cell's structure, function and behaviour. A gene is transcribed (copied) into mRNA (messenger ribonucleic acid), which is then translated (processed) into amino acids (protein building blocks). Only the exons code for the amino acids, so the introns (noncoding regions) are spliced (removed) during transcription. Exon skipping is when one exon is spliced along with the introns in order to counteract a frameshift deletion, where a very small part of the gene is accidentally deleted, causing the exons to shift out of alignment and produce an abnormal protein.
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