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 Bertha (Motor Neuron Disease) at the genomic and sub-genomic levels. Most studies have overlooked association between gene expression and its regulation, which we envisage conducting in order to understand the role of genes that are impaired in Bertha and their master regulators- the small molecules called micro-RNAs. 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 Bertha, and guide us to actual targets that can be exploited in developing treatments. Furthermore, a detailed comparison against myriad of databases will provide additional correlations that are necessary in defining treatments that are global in nature, and have the potential to provide benefit to the community at large.
The uniqueness of our genome work lies in a unidirectional focus and determination 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.