Perspectives on models of spinal muscular atrophy for drug discovery

Matthew E.R. Butchbach; Arthur H.M. Burghes

doi:10.1016/j.ddmod.2004.07.001

Perspectives on models of spinal muscular atrophy for drug discovery

Matthew E.R. Butchbach
, Arthur H.M. Burghes

Ohio State University

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

17 Scopus citations

Abstract

Spinal muscular atrophy (SMA) is a progressive motor neuron disease, which is one of the most common forms of inheritable infant death in the world. SMA results from reduced levels of SMN protein. Various models systems used in SMA research are available and it is possible to assess their strengths and weaknesses. Although each of these models has its limitations, they must be used together so as to effectively and efficiently identify potential SMA therapeutics.

Original language	English
Pages (from-to)	151-156
Number of pages	6
Journal	Drug Discovery Today: Disease Models
Volume	1
Issue number	2
DOIs	https://doi.org/10.1016/j.ddmod.2004.07.001
State	Published - Nov 2004
Externally published	Yes

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10.1016/j.ddmod.2004.07.001

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title = "Perspectives on models of spinal muscular atrophy for drug discovery",

abstract = "Spinal muscular atrophy (SMA) is a progressive motor neuron disease, which is one of the most common forms of inheritable infant death in the world. SMA results from reduced levels of SMN protein. Various models systems used in SMA research are available and it is possible to assess their strengths and weaknesses. Although each of these models has its limitations, they must be used together so as to effectively and efficiently identify potential SMA therapeutics.",

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AU - Burghes, Arthur H.M.

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N2 - Spinal muscular atrophy (SMA) is a progressive motor neuron disease, which is one of the most common forms of inheritable infant death in the world. SMA results from reduced levels of SMN protein. Various models systems used in SMA research are available and it is possible to assess their strengths and weaknesses. Although each of these models has its limitations, they must be used together so as to effectively and efficiently identify potential SMA therapeutics.

AB - Spinal muscular atrophy (SMA) is a progressive motor neuron disease, which is one of the most common forms of inheritable infant death in the world. SMA results from reduced levels of SMN protein. Various models systems used in SMA research are available and it is possible to assess their strengths and weaknesses. Although each of these models has its limitations, they must be used together so as to effectively and efficiently identify potential SMA therapeutics.

UR - https://www.scopus.com/pages/publications/15544385728

U2 - 10.1016/j.ddmod.2004.07.001

DO - 10.1016/j.ddmod.2004.07.001

M3 - Review article

AN - SCOPUS:15544385728

SN - 1740-6757

VL - 1

SP - 151

EP - 156

JO - Drug Discovery Today: Disease Models

JF - Drug Discovery Today: Disease Models

IS - 2

ER -

Perspectives on models of spinal muscular atrophy for drug discovery

Abstract

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