Anti Sense Therapy And The Treatment Of Alzheimer 's Disease And Spinal Muscular Atrophy

1695 Words Nov 7th, 2016 7 Pages
Although Anti-sense Therapy has limitations related to targeted drug delivery, it is still considered as one of the promising technology for treating most of the Rare and Inherited disorders, being categorized as precision medicine has advanced very much with recent advances in drug delivery technologies like lipid nanoparticle (LNP) formulations, cell-targeting technologies. Anti-sense drugs are seen as most potential drugs for treating debilitating conditions with more targeted approach. The journey of Anti-sense oligonucleotides from the state of highly potential drug candidates to a sudden debacle with limitations in drug delivery and toxicity and the resume of these candidates with technological advancements will be reviewed with a special focus on application of this technology for the treatment of Huntington’s disease and spinal muscular atrophy. The most recently accepted application of Nusinersen, an anti-sense oligonucleotide drug for spinal muscular atrophy by US FDA and EMA and many candidates in late stage clinical trials proves the viability and glory of these candidates as potential drugs for treatment of rare diseases and conditions that need more targeted approach.

2) Anti-Sense Therapy:

a) Definition: Anti-sense therapy involves the use of antisense DNA or RNA for the inhibition of transcription or translation of a specific gene or gene product for therapeutic purposes. Anti-Sense oligonucleotides are small pieces of DNA or RNA that can bind to specific…

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