Molecular Targets : From Pathogenesis to Therapeutics

Malfunctioning of cellular and molecular signaling machinery is the cause of many human diseases, and disturbances in the signaling processes and the proteins involved in the control layers are the essential elements in the neurodegenerative disorders. The study of neurological disease at the molecular level has disclosed a diverse array of molecular targets such as gene, protein, DNA, RNA, mi-RNA, etc. and has delineated their contribution to synaptic function.

For instances For instances Ataxia with isolated vitamin E deficiency (AVED) is a neuro-degenerative disease caused by a mutation in the α-tocopherol transfer protein gene (TTPA). Mutation of the androgen receptor gene is the main cause for Kennedy's disease which is also called "X-linked recessive spinal and bulbar muscular atrophy" or "bulbospinal neuronopathy”. Role of aquaporin-1 and aquaporin-4 (AQP1 and AQP4) have been determined in the human central pontine myelinolysis. Several single-nucleotide variants (SNVs) in low-density lipoprotein receptor-related protein 6 (Lrp6) cause neural tube defects and spina bifida (NTDs)s. Sequestration of DROSHA and DGCR8 by expanded (CGG) RNA repeats alters microRNA processing in fragile X-associated tremor/ataxia syndrome's. Very interestingly The mutation in the SOD1 gene and hexa-nucleotide repeat expansion in C9orf72 gene have been known to cause frontotemporal dementia and amyotrophic lateral sclerosis (c9FTD/ALS). Recently A small molecule 1a has been discovered that can work as lead molecule for targeting the r(GGGGCC)exp RNA.

These examples clearly say that, due to advancement in the molecular techniques, the list of these molecular targets and the small molecule that have been used to modulate these targets are increasing day by day. These experimental data are very un-organized and thus tough to predict structure-activity relationship (SAR) in a user-friendly manner.

Therefore it has become very important to collect all these information on a single platform that gives the comprehensive details about these molecular targets and also provides the information about the small molecule which has been studied as a therapeutic for the treatment of these neurological diseases. This collected data on the single platform will lead to the better prediction of structure-activity relationship and open the new dimension of therapeutic development for combating these deadly diseases.