Two articles published in Drug Discovery Today: Therapeutic Strategies highlight current advances in the development of antisense oligonucleotides (AONs) as personalized medicine for the treatment of patients with Duchenne muscular dystrophy (DMD). DMD is a severe X-linked neuromuscular disease whose symptoms can occur as early as 2 years of age and survival to adulthood is extremely rare. This disease is caused by mutations in dystrophin, a gene critical for muscle fiber strength, resulting in a severe reduction of dystrophin protein in muscles. A milder form of DMD is Becker muscular dystrophy (BMD), caused by different mutations in the same gene that preserves some function of the protein. Analyzing the differential consequences of distinct mutations on the same gene, the researchers used antisense oligonucleotides to skipping exon 51 of the dystrophin gene (as in BMD) and that help preserve some of the protein functions (observed in BMD patients) in DMD patients. The authors discuss the preclinical development of two antisense therapies, drisapersen and eteplirsen, two chemically distinct drug candidates that have demonstrated features of exon 51 skipping. The authors believe in the potential of the two drugs, although both have \"advantages and disadvantages regarding safety and pharmacokinetics.\" The authors point out that \"clinical trials of orphan drugs are required to increase the low prevalence of mutations, and the advent of AON technology along with ongoing developments may make personalized medicine for these patients a reality.\"<\/p>","protected":false},"excerpt":{"rendered":"
Due articoli pubblicati su Drug Discovery Today: Therapeutic Strategies evidenziano gli attuali progressi nello sviluppo di oligonucleotidi antisenso (AON) come medicina personalizzata per il trattamento dei pazienti affetti da distrofia muscolare di Duchenne (DMD). La DMD \u00e8 una grave malattia neuromuscolare legata all\u2019X, i cui sintomi possono insorgere gi\u00e0 all\u2019et\u00e0 di 2 anni e la […]<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[1],"tags":[],"class_list":["post-1028","post","type-post","status-publish","format-standard","hentry","category-senza-categoria"],"yoast_head":"\n