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Current Genomics


ISSN (Print): 1389-2029
ISSN (Online): 1875-5488

Identification of Pharmacological Targets in Amyotrophic Lateral Sclerosis Through Genomic Analysis of Deregulated Genes and Pathways

Author(s): Sabrina Paratore, Salvatore Pezzino and Sebastiano Cavallaro

Volume 13, Issue 4, 2012

Page: [321 - 333] Pages: 13

DOI: 10.2174/138920212800793366

Price: $65


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

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