Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

Research output: Contribution to journal › Journal article › Research › peer-review

Standard

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. / Southwell, Amber L; Skotte, Niels H; Bennett, C Frank; Hayden, Michael R.

In: Trends in Molecular Medicine, Vol. 18, No. 11, 11.2012, p. 634-43.

Research output: Contribution to journal › Journal article › Research › peer-review

Harvard

Southwell, AL, Skotte, NH, Bennett, CF & Hayden, MR 2012, 'Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases', Trends in Molecular Medicine, vol. 18, no. 11, pp. 634-43. https://doi.org/10.1016/j.molmed.2012.09.001

APA

Southwell, A. L., Skotte, N. H., Bennett, C. F., & Hayden, M. R. (2012). Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. Trends in Molecular Medicine, 18(11), 634-43. https://doi.org/10.1016/j.molmed.2012.09.001

Vancouver

Southwell AL, Skotte NH, Bennett CF, Hayden MR. Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. Trends in Molecular Medicine. 2012 Nov;18(11):634-43. https://doi.org/10.1016/j.molmed.2012.09.001

Author

Southwell, Amber L ; Skotte, Niels H ; Bennett, C Frank ; Hayden, Michael R. / Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. In: Trends in Molecular Medicine. 2012 ; Vol. 18, No. 11. pp. 634-43.

Bibtex

@article{bb55546722f545f4851a31cd2b8b23fa,
title = "Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases",
abstract = "The rising median age of our population and the age-dependent risk of neurodegeneration translate to exponentially increasing numbers of afflicted individuals in the coming years. Although symptomatic treatments are available for some neurodegenerative diseases, most are only moderately efficacious and are often associated with significant side effects. The development of small molecule, disease-modifying drugs has been hindered by complex pathogenesis and a failure to clearly define the rate-limiting steps in disease progression. An alternative approach is to directly target the mutant gene product or a defined causative protein. Antisense oligonucleotides (ASOs) - with their diverse functionality, high target specificity, and relative ease of central nervous system (CNS) delivery - are uniquely positioned as potential therapies for neurological diseases. Here we review the development of ASOs for the treatment of inherited neurodegenerative diseases.",
keywords = "Animals, Clinical Trials as Topic, Gene Transfer Techniques, Genetic Therapy, Heredodegenerative Disorders, Nervous System, Humans, Oligonucleotides, Antisense, RNA Interference",
author = "Southwell, {Amber L} and Skotte, {Niels H} and Bennett, {C Frank} and Hayden, {Michael R}",
note = "Copyright {\textcopyright} 2012 Elsevier Ltd. All rights reserved.",
year = "2012",
month = nov,
doi = "10.1016/j.molmed.2012.09.001",
language = "English",
volume = "18",
pages = "634--43",
journal = "Trends in Molecular Medicine",
issn = "1471-4914",
publisher = "Elsevier Ltd. * Trends Journals",
number = "11",

}

RIS

TY - JOUR

T1 - Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

AU - Southwell, Amber L

AU - Skotte, Niels H

AU - Bennett, C Frank

AU - Hayden, Michael R

N1 - Copyright © 2012 Elsevier Ltd. All rights reserved.

PY - 2012/11

Y1 - 2012/11

N2 - The rising median age of our population and the age-dependent risk of neurodegeneration translate to exponentially increasing numbers of afflicted individuals in the coming years. Although symptomatic treatments are available for some neurodegenerative diseases, most are only moderately efficacious and are often associated with significant side effects. The development of small molecule, disease-modifying drugs has been hindered by complex pathogenesis and a failure to clearly define the rate-limiting steps in disease progression. An alternative approach is to directly target the mutant gene product or a defined causative protein. Antisense oligonucleotides (ASOs) - with their diverse functionality, high target specificity, and relative ease of central nervous system (CNS) delivery - are uniquely positioned as potential therapies for neurological diseases. Here we review the development of ASOs for the treatment of inherited neurodegenerative diseases.

AB - The rising median age of our population and the age-dependent risk of neurodegeneration translate to exponentially increasing numbers of afflicted individuals in the coming years. Although symptomatic treatments are available for some neurodegenerative diseases, most are only moderately efficacious and are often associated with significant side effects. The development of small molecule, disease-modifying drugs has been hindered by complex pathogenesis and a failure to clearly define the rate-limiting steps in disease progression. An alternative approach is to directly target the mutant gene product or a defined causative protein. Antisense oligonucleotides (ASOs) - with their diverse functionality, high target specificity, and relative ease of central nervous system (CNS) delivery - are uniquely positioned as potential therapies for neurological diseases. Here we review the development of ASOs for the treatment of inherited neurodegenerative diseases.

KW - Animals

KW - Clinical Trials as Topic

KW - Gene Transfer Techniques

KW - Genetic Therapy

KW - Heredodegenerative Disorders, Nervous System

KW - Humans

KW - Oligonucleotides, Antisense

KW - RNA Interference

U2 - 10.1016/j.molmed.2012.09.001

DO - 10.1016/j.molmed.2012.09.001

M3 - Journal article

C2 - 23026741

VL - 18

SP - 634

EP - 643

JO - Trends in Molecular Medicine

JF - Trends in Molecular Medicine

SN - 1471-4914

IS - 11

ER -

ID: 153451293