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Antisense oligonucleotides: A primer.

Abstract

There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine. Indeed, existing pipelines for the development of ASO therapies for spinocerebellar ataxias, Huntington disease, Alzheimer disease, amyotrophic lateral sclerosis, Parkinson disease, and others, and increased focus by the pharmaceutical industry on ASO development, strengthen the outlook for using ASOs for neurodegenerative diseases. Perhaps the most significant advantage to ASO therapeutics over other small molecule approaches is that acquisition of the target sequence provides immediate knowledge of putative complementary oligonucleotide therapeutics. In this review, we describe the various types of ASOs, how they are used therapeutically, and the present efforts to develop new ASO therapies that will contribute to a forthcoming toolkit for treating multiple neurodegenerative diseases.

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  • Authors+Show Affiliations

    ,

    Department of Neurology (D.R.S., S.M.P.), University of Utah, Salt Lake City, UT; and Center for the Science of Therapeutics (E.V.M.), Broad Institute of MIT and Harvard, Cambridge, MA.

    ,

    Department of Neurology (D.R.S., S.M.P.), University of Utah, Salt Lake City, UT; and Center for the Science of Therapeutics (E.V.M.), Broad Institute of MIT and Harvard, Cambridge, MA.

    Department of Neurology (D.R.S., S.M.P.), University of Utah, Salt Lake City, UT; and Center for the Science of Therapeutics (E.V.M.), Broad Institute of MIT and Harvard, Cambridge, MA.

    Source

    Neurology. Genetics 5:2 2019 Apr pg e323

    Pub Type(s)

    Journal Article
    Review

    Language

    eng

    PubMed ID

    31119194

    Citation

    Scoles, Daniel R., et al. "Antisense Oligonucleotides: a Primer." Neurology. Genetics, vol. 5, no. 2, 2019, pp. e323.
    Scoles DR, Minikel EV, Pulst SM. Antisense oligonucleotides: A primer. Neurol Genet. 2019;5(2):e323.
    Scoles, D. R., Minikel, E. V., & Pulst, S. M. (2019). Antisense oligonucleotides: A primer. Neurology. Genetics, 5(2), pp. e323. doi:10.1212/NXG.0000000000000323.
    Scoles DR, Minikel EV, Pulst SM. Antisense Oligonucleotides: a Primer. Neurol Genet. 2019;5(2):e323. PubMed PMID: 31119194.
    * Article titles in AMA citation format should be in sentence-case
    TY - JOUR T1 - Antisense oligonucleotides: A primer. AU - Scoles,Daniel R, AU - Minikel,Eric V, AU - Pulst,Stefan M, Y1 - 2019/04/01/ PY - 2018/12/17/received PY - 2019/02/14/accepted PY - 2019/5/24/entrez PY - 2019/5/24/pubmed PY - 2019/5/24/medline SP - e323 EP - e323 JF - Neurology. Genetics JO - Neurol Genet VL - 5 IS - 2 N2 - There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine. Indeed, existing pipelines for the development of ASO therapies for spinocerebellar ataxias, Huntington disease, Alzheimer disease, amyotrophic lateral sclerosis, Parkinson disease, and others, and increased focus by the pharmaceutical industry on ASO development, strengthen the outlook for using ASOs for neurodegenerative diseases. Perhaps the most significant advantage to ASO therapeutics over other small molecule approaches is that acquisition of the target sequence provides immediate knowledge of putative complementary oligonucleotide therapeutics. In this review, we describe the various types of ASOs, how they are used therapeutically, and the present efforts to develop new ASO therapies that will contribute to a forthcoming toolkit for treating multiple neurodegenerative diseases. SN - 2376-7839 UR - https://www.unboundmedicine.com/medline/citation/31119194/Antisense_oligonucleotides:_A_primer L2 - http://ovidsp.ovid.com/ovidweb.cgi?T=JS&PAGE=linkout&SEARCH=31119194.ui DB - PRIME DP - Unbound Medicine ER -