Abstract
Amyotrophic Lateral Sclerosis (ALS) is a progressive and disabling neurodegenerative disorder characterized by upper and lower motor neuron loss, leading to respiratory insufficiency and death after 3-5 years. Riluzole is currently the only FDA approved drug for ALS, but it has only modest effects on survival. The majority of ALS cases are sporadic and probably associated to a multifactorial etiology. With the completion of genome sequencing in humans and model organisms, together with the advent of DNA microarray technology, the transcriptional cascades and networks underlying neurodegeneration in ALS are being elucidated providing new potential pharmacological targets. The main challenge now is the effective screening of the myriad of targets to identify those with the most therapeutic utility. The present review will illustrate how the identification, prioritization and validation of preclinical therapeutics can be achieved through genomic analysis of critical pathways and networks deregulated in ALS pathology.
Keywords: ALS, drug, pathway, pharmacogenomics, networks, target, DNA microarray technology, myriad, neurodegenerative, sporadic, genomic analysis
Current Genomics
Title:Identification of Pharmacological Targets in Amyotrophic Lateral Sclerosis Through Genomic Analysis of Deregulated Genes and Pathways
Volume: 13 Issue: 4
Author(s): Sabrina Paratore, Salvatore Pezzino and Sebastiano Cavallaro
Affiliation:
Keywords: ALS, drug, pathway, pharmacogenomics, networks, target, DNA microarray technology, myriad, neurodegenerative, sporadic, genomic analysis
Abstract: Amyotrophic Lateral Sclerosis (ALS) is a progressive and disabling neurodegenerative disorder characterized by upper and lower motor neuron loss, leading to respiratory insufficiency and death after 3-5 years. Riluzole is currently the only FDA approved drug for ALS, but it has only modest effects on survival. The majority of ALS cases are sporadic and probably associated to a multifactorial etiology. With the completion of genome sequencing in humans and model organisms, together with the advent of DNA microarray technology, the transcriptional cascades and networks underlying neurodegeneration in ALS are being elucidated providing new potential pharmacological targets. The main challenge now is the effective screening of the myriad of targets to identify those with the most therapeutic utility. The present review will illustrate how the identification, prioritization and validation of preclinical therapeutics can be achieved through genomic analysis of critical pathways and networks deregulated in ALS pathology.
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Cite this article as:
Paratore Sabrina, Pezzino Salvatore and Cavallaro Sebastiano, Identification of Pharmacological Targets in Amyotrophic Lateral Sclerosis Through Genomic Analysis of Deregulated Genes and Pathways, Current Genomics 2012; 13 (4) . https://dx.doi.org/10.2174/138920212800793366
DOI https://dx.doi.org/10.2174/138920212800793366 |
Print ISSN 1389-2029 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5488 |
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