Antisense drugs target amyotrophic lateral sclerosis
Antisense drugs are gene-based molecules that inhibit the synthesis of proteins (including proteins that cause specific diseases) by binding to the ribonucleic acids (RNAs) responsible for their formation. Specifically, these drugs are single-stranded short polymers of RNA or deoxyribonucleic acid (DNA), termed oligonucleotides, designed to contain part of the noncoding strand of messenger RNA (mRNA), which is a molecule involved in translating DNA into protein. Antisense medications are therefore capable of hybridizing with and inactivating the mRNA, preventing the associated gene from producing the unwanted protein. With their anticancer, antiviral, and anti-inflammatory therapeutic capacities, these drugs have been applied in the treatment of various genetic disorders and infections, including diabetes, rheumatoid arthritis, cytomegalovirus retinitis (a virally caused form of blindness that occurs often in AIDS patients), asthma, hypercholesterolemia (a genetic derangement of fat metabolism characterized by very high levels of cholesterol in the blood), and numerous cancers. Research is also being conducted on patients suffering from Parkinson's disease and Huntington's disease to determine whether antisense therapy can mitigate the effects of these conditions. See also: Biotechnology; Deoxyribonucleic acid (DNA); Disease; Gene; Genetic engineering; Oligonucleotide; Protein; Ribonucleic acid (RNA)