Apr 9, 2015

Selection of Potential Pharmacological Targets in ALS Based on Whole- Genome Expression Profiling

Current Medicinal Chemistry
Giovanna MorelloSebastiano Cavallaro

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal disease caused by the gradual degeneration and death of upper and lower motor neurons. Despite continue efforts, the etiology and pathogenesis of ALS are not well understood yet. The lack of knowledge about molecular and cellular players involved in the neurodegenerative progression of ALS hinders effective therapy development. Several genomicbased studies have been conducted to identify genetic contributors to sporadic ALS (SALS) and new potential pharmacological targets, but these have resulted in short and non-overlapping lists of candidates. In the last few years, our research group has developed the largest whole-genome expression profile database of SALS human samples. We have identified several genes deregulated in the motor cortex of SALS patients and analyzed the role of these genes within deregulated pathways, providing a full molecular portrait of ALS pathogenesis. Some of deregulated genes encode for proteins that are direct or indirect targets of experimental or therapeutic drugs already applied to unrelated diseases. In this review, we focus on the potential role of candidate targets in ALS pathophysiology, highlighting their possible contribution to ALS therapy. The ...Continue Reading

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Mentioned in this Paper

Study
Pathogenic Aspects
Biochemical Pathway
Research
Abnormal Degeneration
Pathogenesis
Genome
Genes
Upper
Cessation of Life

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