Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets

Joseph R. Klim; Caroline Vance; Emma L. Scotter

doi:10.1016/j.biocel.2019.03.009

Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets

Joseph R. Klim, Caroline Vance, Emma L. Scotter^*

^*Corresponding author for this work

Basic and Clinical Neuroscience

Research output: Contribution to journal › Short survey › peer-review

22 Citations (Scopus)

726 Downloads (Pure)

Abstract

Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic.

Original language	English
Pages (from-to)	149-153
Number of pages	5
Journal	International Journal of Biochemistry and Cell Biology
Volume	110
Early online date	20 Mar 2019
DOIs	https://doi.org/10.1016/j.biocel.2019.03.009
Publication status	Published - May 2019

Keywords

Amyotrophic lateral sclerosis
Antisense oligonucleotides
Molecular medicine
Neurodegenerative diseases

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10.1016/j.biocel.2019.03.009

Antisense oligonucleotide therapies for_KIM_Acc20Mar2019Acc20Mar2019Accepted author manuscript, 997 KBLicence: CC BY-NC-ND

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title = "Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets",

abstract = "Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic.",

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T1 - Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis

T2 - Existing and emerging targets

AU - Klim, Joseph R.

AU - Vance, Caroline

AU - Scotter, Emma L.

PY - 2019/5

Y1 - 2019/5

N2 - Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic.

AB - Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic.

KW - Amyotrophic lateral sclerosis

KW - Antisense oligonucleotides

KW - Molecular medicine

KW - Neurodegenerative diseases

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DO - 10.1016/j.biocel.2019.03.009

M3 - Short survey

C2 - 30904737

AN - SCOPUS:85063318204

SN - 1357-2725

VL - 110

SP - 149

EP - 153

JO - International Journal of Biochemistry and Cell Biology

JF - International Journal of Biochemistry and Cell Biology

ER -

Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets

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