AAN.com
Quick search in Citations
AnywhereCitation
Advanced Search
Trending Terms:
Views and Reviews
April 1, 2019
Open Access

Antisense oligonucleotides
A primer

Daniel R. Scoles, PhD, Eric V. Minikel, MS, and Stefan M. Pulst, MD, Dr medAuthors Info & Affiliations
April 2019 issue
5 (2)
https://doi.org/10.1212/NXG.0000000000000323
Letters to the Editor
Track CitationsAdd to favorites
Full Text HTML

Information & Authors

Information

Published In

Neurology® Genetics
Volume 5 • Number 2 • April 2019

Copyright

Copyright © 2019 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

Publication History

Received: December 17, 2018
Accepted: February 14, 2019
Published online: April 1, 2019
Published in print: April 2019

Permissions

Request permissions for this article.

Disclosure

D.R. Scoles has received research support from the NINDS (U01NS103883 and R01NS097903) and Harrington Discovery Institute. E.V. Minikel has received funding for travel and/or speaker honoraria from Illumina and has received research support from Charles River Laboratories, NIH (F31 AI22952), and Prion Alliance. S.M. Pulst serves on the editorial boards of Journal of Cerebellum, NeuroMolecular Medicine, Experimental Neurology, Neurogenetics, Nature Clinical Practice, and Neurology: Genetics; holds patents for Nucleic acids encoding ataxin-2 binding proteins; Nucleic acid encoding Schwannomin-binding proteins and products related thereto; Transgenic mouse expressing a polynucleotide encoding a human ataxin-2 polypeptide; Methods of detecting SCA-2 nucleic acids; Nucleic acid encoding SCA-2 and products related thereto; Schwannomin-binding-proteins; and Compositions and methods for SCA; has received publishing royalties for The Ataxias (Churchill Livingston, 2007), Genetics in Neurology (ANN Press, 2005), Genetics of Movement Disorders (Academic Press, 2003), Neurogenetics (Oxford University Press, 2000), and Molecular Genetic Testing in Neurology, 2nd to 5th (AAN Press, 1996); serves or has served as a consultant for Ataxion Therapeutics; has received research support from the NIH (RC1NS068897, RC4NS073009, R21NS081182, R21NS079852, and R01NS33123) and the National Ataxia foundation; and has received license fee payments for technology or inventions from the Cedars-Sinai Medical Center. Disclosures available: Neurology.org/NG.

Study Funding

No targeted funding reported.

Authors

Affiliations & Disclosures

Daniel R. Scoles, PhD
From the 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.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
NONE
Editorial Boards:
1.
NONE
Patents:
1.
NONE
Publishing Royalties:
1.
NONE
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
NONE
Speakers' Bureaus:
1.
NONE
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
NONE
Research Support, Government Entities:
1.
NINDS, U01 NS103883, PI, 1 yr to date. NINDS, R01 NS097903, PI, 1 yr to date.
Research Support, Academic Entities:
1.
NONE
Research Support, Foundations and Societies:
1.
Harrington Discovery Institute, Harrington Rare Disease Scholar.
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
NONE
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE
View all articles by this author
Eric V. Minikel, MS
From the 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.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
Illumina, speaker honorarium
Editorial Boards:
1.
NONE
Patents:
1.
NONE
Publishing Royalties:
1.
NONE
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
NONE
Speakers' Bureaus:
1.
NONE
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
Charles River Laboratories
Research Support, Government Entities:
1.
NIH, F31 AI22952, PI, 2016-2019
Research Support, Academic Entities:
1.
NONE
Research Support, Foundations and Societies:
1.
Prion Alliance
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
NONE
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE
View all articles by this author
Stefan M. Pulst, MD, Dr med
From the 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.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
NONE
Editorial Boards:
1.
NONE
Patents:
1.
Nucleic acids encoding ataxin-2 binding proteins; Nucleic acid encoding Schwannomin-binding-proteins and products related thereto; Transgenic mouse expressing a polynucleotide encoding a human ataxin-2 polypeptide; Methods of detecting spinocerebellar ataxia-2 nucleic acids; Nucleic acid encoding spinocerebellar ataxia-2 and products related thereto; Shwannomin-binding-proteins; Compositions and methods for spinocerebellar ataxia
Publishing Royalties:
1.
The Ataxias (Churchill Livingston, 2007), Genetics in Neurology (ANN Press, 2005), Genetics of Movement Disorders (Academic Press, 2003), Neurogenetics (Oxford University Press, 2000), Molecular Genetic Testing in Neurology, 2nd - 5th (AAN Press, 1996)
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
Ataxion Therapeutics
Speakers' Bureaus:
1.
Athena Diagnostics, Inc.
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
NONE
Research Support, Government Entities:
1.
(1) 2010-2012 National Institutes of Health (RC1NS068897): CLINICAL RESEARCH CONSORTIUM FOR SPINOCEREBELLAR ATAXIAS, 9/1/2009-8/31/2012 (Principal Investigator of genomics core and site PI). (2) 2010-2013 National Institutes of Health (RC4NS073009): Drug discovery for Spinocerebellar ataxia type 2 (SCA2). 9/1/2010-8/31/2013 (Principle Investigator, Co-PI, D. Scoles). (3) 2013-2015 National Institutes of Health (R21NS081182): Antisense oligonucleotides for the treatment of spinocerebellar ataxia type 2. 07/01/2013 to 06/30/2015 (Co- PI with D. Scoles). (4) 2013-2016 National Institutes of Health (R21NS079852): Identification of a mutation causing Purkinje cell degeneration in the rat. 03/01/2013 to 02/28/2016. (Principle Investigator). (5) 2014-2021 National Institutes of Health (RO1NS33123): Spinocerebellar ataxia type 2: gene and gene product. 9/30/14-6/30/2021. (Principal Investigator).
Research Support, Academic Entities:
1.
NONE
Research Support, Foundations and Societies:
1.
National Ataxia foundation (mentor for fellowship award)
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
Cedars-Sinai Medical Center
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE
View all articles by this author

Notes

Correspondence Dr. Scoles [email protected] or Dr. Pulst [email protected]
Funding information and disclosures are provided at the end of the article. Full disclosure form information provided by the authors is available with the full text of this article at Neurology.org/NG.
The Article Processing Charge was waived at the discretion of the Editor.

Author Contributions

D.R. Scoles wrote the manuscript and produced the figures. E.V. Minikel further contributed to ASO chemistry. S.M. Pulst further contributed to components on movement disorders and neuromuscular diseases.

Metrics & Citations

Metrics

Citations

Download Citations

If you have the appropriate software installed, you can download article citation data to the citation manager of your choice. Select your manager software from the list below and click Download.

Cited By
    • Ariel Solis Chiriboga,
    • Alberto Bustillos,
    Review of the Use of Antisense Oligonucleotides as Therapy for Huntington's Disease, Salud, Ciencia y Tecnología - Serie de Conferencias, 3, (923), (2024).https://doi.org/10.56294/sctconf2024923
    Crossref
    • Ariel Solis-Chiriboga,
    • Alberto Bustillos,
    Use of Antisense Oligonucleotides as Therapy for Huntington's Disease, Salud, Ciencia y Tecnología - Serie de Conferencias, 3, (795), (2024).https://doi.org/10.56294/sctconf2024795
    Crossref
    • Andrew N. Lane,
    • Richard M. Higashi,
    • Teresa W-M. Fan,
    Challenges of Spatially Resolved Metabolism in Cancer Research, Metabolites, 14, 7, (383), (2024).https://doi.org/10.3390/metabo14070383
    Crossref
    • Jelisah F. Desgraves,
    • Mynor J. Mendez Valdez,
    • Jay Chandar,
    • Muhammet Enes Gurses,
    • Lisa Henderson,
    • Jesus R. Castro,
    • Deepa Seetheram,
    • Michael E. Ivan,
    • Ricardo J. Komotar,
    • Ashish H. Shah,
    Antisense Oligonucleotides for Rapid Translation of Gene Therapy in Glioblastoma, Cancers, 16, 10, (1944), (2024).https://doi.org/10.3390/cancers16101944
    Crossref
    • Clara Mayer,
    • Lluís Riera-Ponsati,
    • Sakari Kauppinen,
    • Henrik Klitgaard,
    • Janine T. Erler,
    • Stine N. Hansen,
    Targeting the NRF2 pathway for disease modification in neurodegenerative diseases: mechanisms and therapeutic implications, Frontiers in Pharmacology, 15, (2024).https://doi.org/10.3389/fphar.2024.1437939
    Crossref
    • Aidan J. Murphy,
    • Steve D. Wilton,
    • May T. Aung-Htut,
    • Craig S. McIntosh,
    Down syndrome and DYRK1A overexpression: relationships and future therapeutic directions, Frontiers in Molecular Neuroscience, 17, (2024).https://doi.org/10.3389/fnmol.2024.1391564
    Crossref
    • Zahra Javanmard,
    • Maryam Pourhajibagher,
    • Abbas Bahador,
    Characteristics and Applications of Peptide Nucleic Acid in the Treatment of Infectious Diseases and the Effect of Antimicrobial Photodynamic Therapy on Treatment Effectiveness, Infectious Disorders - Drug Targets, 24, 1, (2024).https://doi.org/10.2174/1871526523666230724120957
    Crossref
    • Barney Viengkhou,
    • Christine Hong,
    • Curt Mazur,
    • Sagar Damle,
    • Nicholas B. Gallo,
    • Terry C. Fang,
    • Kate Henry,
    • Iain L. Campbell,
    • Fredrik Kamme,
    • Markus J. Hofer,
    Interferon-α receptor antisense oligonucleotides reduce neuroinflammation and neuropathology in a mouse model of cerebral interferonopathy, Journal of Clinical Investigation, 134, 4, (2024).https://doi.org/10.1172/JCI169562
    Crossref
    • Jenna Gale,
    • Elias Aizenman,
    The physiological and pathophysiological roles of copper in the nervous system, European Journal of Neuroscience, 60, 1, (3505-3543), (2024).https://doi.org/10.1111/ejn.16370
    Crossref
    • Dragana Protic,
    • Randi Hagerman,
    State‐of‐the‐art therapies for fragile X syndrome, Developmental Medicine & Child Neurology, 66, 7, (863-871), (2024).https://doi.org/10.1111/dmcn.15885
    Crossref
  11. See more

View Options

View options

PDF and All Supplements

Download PDF and Supplementary Material

Full Text HTML

View Full Text HTML
Login options

Check if you have access through your login credentials or your institution to get full access on this article.

Personal login Institutional Login
Purchase Options

The neurology.org payment platform is currently offline. Our technical team is working as quickly as possible to restore service.

If you need immediate support or to place an order, please call or email customer service:

  • 1-800-638-3030 for U.S. customers - 8:30 - 7 pm ET (M-F)
  • 1-301-223-2300 for customers outside the U.S. - 8:30 - 7 pm ET (M-F)
  • [email protected]

We appreciate your patience during this time and apologize for any inconvenience.

Media

Figures

Other

Tables

Share

Share

Share article link

Share

View full text|Download PDF