THE IMPORTANCE AND VALUE OF THE GENOME
The story of our life is imprinted in the genome, then why we don’t look at the genome to unveil the secrets of neurodegenerative diseases. We are living in an era where we can now sequence the whole human genome (>3 billion base pairs) in quick time. The disease programming takes place in the genome, therefore, the genome carries vital information about a disease that we can’t see from the outside, and can guide us not only in understanding the disease, but also in defining future treatments, diagnostic and prognostic modalities.
We are currently studying Motor Neuron Disease and other NDs at the genomic and sub-genomic levels. Most studies have overlooked association between gene expression and its regulation, which we have taken on board as our integral approach for understanding how genes are regulated by miRNAs. With this knowledge, we plan to design inhibitors to normalise gene function, create novel therapeutic modalities based on micro-RNA targeting, and provide possible curative strategies. This novel approach can guide us in designing precision or tailored treatments that can be individualized to patient’s need. We see the biggest chances of success because of the holistic and in-depth nature of our approach.
Complete mapping of the whole genomes and their regulators will provide a clear genomic and sub-genomic understanding of the pathways that impinge on NDs, and guide us to actual targets that can be exploited in developing treatments. Furthermore, a detailed comparison against a myriad of databases will provide additional global perspective on correlations that are necessary in defining treatments, and have the potential to provide benefit to the community at large.
The uniqueness of our genome work lies in a unidirectional focus and multi-entity determination of MND and NDs in providing design and development of new generation of genome-based therapies, biomarkers for the treatment, diagnosis, and prognosis, in addition to providing a profound understanding of this disease.