Antisense molecules: A new class of drugs

Abstract

An improved understanding of disease pathogenesis leads to identification of novel therapeutic targets. From a pharmacologic point of view, these can be addressed by small chemical compounds, so-called biologicals (eg, mAbs and recombinant proteins), or by a rather new class of molecule based on the antisense concept. Recently, a new wave of clinical studies exploring antisense strategies is evolving. In addition to cancer, they include predominantly trials on infectious and noninfectious diseases, such as chronic inflammatory and metabolic conditions. This article, based on a systematic PubMed literature search, highlights recent developments in this emerging field.

GATA‐3‐specific DNAzyme — A novel approach for stratified asthma therapy

“…The high level of homology between the different members of the GATA family, especially in the functionally most relevant DNA‐binding region, most likely hindered the selection of inhibitors with sufficiently high selectivity for GATA‐3. Therefore, antisense‐based technologies, which interfere at the level of mRNA expression and that have recently made major advances toward clinical application for other diseases were explored. Alternative antisense approaches and their application specifically for the treatment of respiratory system disorders have most recently been reviewed by Moschos et al.…”

Posibilidades de tratamiento en la atrofia espinal infantil

“…Los oligonucleótidos antisentido son análogos a secuencias específicas de ARN que se unen al pre-ARN y omiten o añaden exones en su transcripción a ARNm [34]. Se acoplan a moléculas diferentes, como fosforotioatos o morfolinos, que les confieren estabilidad, resistencia a endonucleasas y mejoran su penetración en tejidos y células [35].…”

Anti-sense oligonucleotide therapies for the treatment of hyperlipidaemia

“…The newest type of therapeutic intervention relies on nucleic acid chemistry rather than small organic molecules or antibodies [1]. A number of approaches have been developed that include antisense deoxyribonucleic acid (DNA) oligonucleotides, small interfering RNAs (siRNAs) or microRNAs (miRNAs), and enzymes based either on DNA (DNAzymes) or RNA (RNAzymes) ( Table 1) [1,7,8].…”

“…The availability of nucleic-acid-based inhibitors allows precise scientific targeting of genes and intracellular pathways that cannot be achieved by classical smallmolecule inhibitors or antibodies [1]. These have proved useful in clarifying the roles of genes involved in metabolism and especially in exploring the significance of regulatory ribonucleic acids (RNAs).…”