New Components for Antisense Gene Therapy Show Promise in Treating Spinal Muscular Atrophy

Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. Their results may lead to the development of drugs with less toxicity and fewer injections needed thanks to prolonged action. The paper was published in the journal Nucleic Acid Therapeutics.

Antisense oligonucleotides are single stranded chemically modified fragments of DNA that target pre-messenger RNA, short bits of genetic information a ribosome reads to make a protein. Depending on how a particular antisense oligonucleotide works, the target mRNA can either be destroyed or undergo subtle changes in how it’s spliced, i.e. how exons, the coding regions, are excluded or included in the final mRNA.

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