New options for targeting gene mutation in FA described in nucleic acid therapeutics

(Mary Ann Liebert, Inc./Genetic Engineering News) Researchers have shown that a wide variety of synthetic antisense oligonucleotides with different chemical modifications can activate the frataxin gene, which contains a mutation that decreases its expression in the inherited neurologic disorder Friedreich's ataxia (FA).
Source: EurekAlert! - Biology - Category: Biology Source Type: news