Delivery of antisense molecules
After human genome sequencing, great strides have been made towards targeting genomic DNA, mRNA and non-coding RNA (miRNAs). Sequence selective targeting of genetic material by using synthetic nucleic acid analogues remains a holy grail for new drug discovery and development program. Tremendous promising work has been done to improve the biophysical properties of nucleic acid analogues. However, effective nucleic acids delivery strategies need to be devised to expand their biological applications. Our research is focused on devising new delivery strategies for nucleic acids analogues for effective gene therapy.
Site specific delivery of gene correction reagents
Myriads of genetic diseases are caused by point mutations in the genomic DNA. Several macromolecular based bio-therapeutics; meganucleases, ZFNs, TALENS and CRISPR/Cas9 technology have been deployed to wage war against these genetic disorders by provoking DNA repair machinery inside mammalian cells. Though promising results have been revealed at ex vivo level, however, in vivo targeting of these macromolecules and off target toxicity remains an Achilles heel for their clinical applications. Our research will be centered on devising new strategies to deliver gene correcting molecules to site of actions and improving their efficacy.
Targeting tumor microenvironment
Selective tumor targeting has always been an area of interest for successful cancer therapy. Targeting tumor cells selectively without affecting normal bystander cells require unique knowledge at chemistry/biology interface. This research program will be centered on targeting tumor microenvironment selectively using peptide and carbohydrate based scaffolds in combination with nanotechnology.