Humans have always lived with genetic diseases, as these arise from aberrations in our very genome. About 7,000 different genetic diseases have been recorded, with an estimated 700 million people suffering from them worldwide. Till recently, genetic diseases were at the fringes of the healthcare system since we lacked technologies to make correct diagnoses and study the underlying basis of disease. This is rapidly changing, with innovations in next generation DNA sequencing and AI tools improving the accuracy and speed of diagnosis. Advances in stem cell technology are helping to create cellular models to study disease pathology and test therapeutic molecules. Perhaps the most powerful set of tools that could potentially treat a very large number of monogenic diseases (caused by defect in a single gene) are those that operate at the nucleic acid level. These include delivery of a correct copy of the gene, or of its messenger RNA; correcting the mutation by CRISPR/ Cas; or tweaking the splicing or expression of the gene with oligonucleotides. These technologies are already yielding treatments for some rare genetic diseases, with a few approved drugs in the market and many more in clinical trials. However, this area of research is still nascent, with many challenges that need to be overcome. I shall highlight some of these in my talk, with a view to attract more scientists, especially chemists to contribute to this multidisciplinary area. Considering the phenomenal cost of some of the rare disease drugs (Zolgensma, a single dose gene therapy at Rs. 16 crores), it is important for us in India to develop the capability of discovering, optimizing and manufacturing these drugs indigenously.